Precision Medicine, Personalized Care
The Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation
2 Welcome from the Division Chief 4 PIPseq: A Novel Approach to Precision Medicine
A Future with Avatars? Bringing New Therapies to Children with Cancer
8 A Research-Based Approach to Cancer
Teamwork Moves Leukemia Research to the Clinic And Baby Makes Three A Unique Center Focused on a Rare Skin Cancer National Leadership in Hodgkin Lymphoma
12 Innovative Stem Cell Transplantation Special Expertise for a Rare Disease Back to a Life of Dancing and Skating Quality Care — with an Eye on Cost
15 A Holistic Approach to Wellness
Palliative Care from Day One A Special Place for Sibs A Team Approach to One Little Girl's Care Navigating with Words
19 Comprehensive Programs for Nonmalignant Hematologic Disorders
Ensuring Compliance with Sickle Cell Care Can Vitamin D Reduce Pulmonary Complications? A Brother's Gift A Helping Hand — and Heart — for Kids with Sickle Cell
23 Dr. James A. Wolff: A Legacy of Leadership 24 Hope & Heroes
At NewYork-Presbyterian/Morgan Stanley Children’s Hospital at Columbia University Medical Center, our goals are not only to provide effective therapies for malignancies and nonmalignant hematologic disorders, but to return children to lives that are as normal and constructive as possible — so they can grow up to be happy and healthy adults.
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Welcome from the Division Chief
Dr. Kung’s research focuses on identifying new ways to treat a variety of cancers. His laboratory utilizes a cross-disciplinary approach to identify vulnerabilities in different kinds of cancer, and to develop novel treatments that exploit those weaknesses. His research has led to clinical trials of promising new therapies for patients with brain tumors, blood cancers, and sarcomas.
Pediatric hematology and oncology specialists at NewYork-Presbyterian/ Morgan Stanley Children’s Hospital combine cutting-edge expertise with compassion when caring for every patient who walks through our doors. Our goals are not only to provide effective therapies for malignancies and nonmalignant hematologic disorders, but to return children to lives that are as normal and productive as possible, so they can grow up to be happy and healthy adults. Founded in 1959, the Division is one of the oldest and largest centers in the country focused on treating children with cancer and blood disorders. We recognize that a diagnosis of cancer or a blood disorder is a frightening prospect that affects not only the child, but the entire family. While we treat the child’s disease, we keep at the forefront our obligation to care for the child and the family as a whole.
What sets our program apart? • It is rooted in science, with strong laboratory and translational research
initiatives and a robust portfolio of clinical trials, including pivotal early-phase studies. We are combining the fruits of these investigations with advances in genomics and molecular biology to identify more precise methods to treat patients. Key to this approach is our Precision in Pediatric Sequencing (PIPseq) program, in which we are offering full molecular characterization for every pediatric oncology patient treated in our Division.
• We were one of the first members of the Children’s Oncology Group (COG), whose research has led to dramatic improvements in childhood cancer survival rates. Our physicians lead many national clinical trials within COG and other cooperative groups, and are known internationally for their expertise.
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• Our comprehensive sickle cell program provides the most advanced and comprehensive care for patients with sickle cell disease, including access to clinical trials to prevent strokes and other complications. • In addition to the treatment of malignant diseases, our bone marrow transplantation program has pioneered low-toxicity approaches that allow us to offer curative options to patients with malignancies and nonmalignant diseases. • We meet the needs of a diverse group of patients and families and promote access for residents of medically underserved communities.
There have been dramatic advances in the way we manage pediatric cancers and blood disorders, and today patients are reaping the benefits of decades of research.
• We were the first pediatric hematology/oncology program in the United States to integrate complementary medicine into the treatment plan of every young patient, and we have expanded this commitment to patient well-being through the Center for Comprehensive Wellness. • If other medical needs arise — and this is not uncommon during long-term therapy for childhood cancers and blood disorders — our dedicated children’s hospital has all of the pediatric specialists and subspecialists onsite to address those needs. There have been dramatic advances in the way we manage pediatric cancers and blood disorders, and today patients are reaping the benefits of decades of research. I invite you to learn about the strengths of our program. For more information or to refer a patient, please call us at (212) 305-9770. Sincerely,
Andrew Kung, MD, PhD Chief, Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation NewYork-Presbyterian/Morgan Stanley Children’s Hospital Robert and Ellen Kapito Professor of Pediatrics Columbia University College of Physicians and Surgeons
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PIPseq: A Novel Approach to Precision Medicine
With next generation sequencing, we can delve deeper into the genetic basis of cancer to tailor treatments for patients in a more precise manner.
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PIPSEQ PRECISION MEDICINE
PIPseq has the potential to propel pediatric cancer care to new levels of success.
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hile tremendous progress has been made in the treatment of pediatric cancers, oncologists are still challenged by cancers that fail to respond or develop resistance to treatment. The key to our success in the battle against these cancers lies in the biology of each child’s cancer. What mutations are driving its growth, or rendering it invulnerable to the standard therapies we try to use?
Genetic sequencing can be used to identify the molecular drivers of each patient’s cancer — knowledge that can be further applied to personalize treatment
The answers to those questions will increasingly come from our Precision in Pediatric Sequencing (PIPseq) program. “Through this initiative, we’re aiming to identify the molecular drivers of each patient’s cancer and use this information to personalize his or her treatment using novel, biologically targeted investigational agents,” explains Julia Glade Bender, MD, Medical Director of PIPseq. “With next generation sequencing, we can delve deeper into the genetic basis of cancer to pinpoint novel therapeutic targets.” This customized treatment approach is gaining speed across the spectrum of cancer care, including adult cancers, and has the potential to propel pediatric cancer care to new levels of success.
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A Future with Avatars? The creation of “avatar” laboratory models by propagating tumors from patients in mice has been shown to be a promising way to assess the efficacy of potential new drug treatments. NYP/Morgan Stanley Children’s is the only center in New York City routinely applying this technology to pediatric cancers. As part of the PIPseq program, our investigators are creating patient-derived xenograft models (human tumors propagated in mice) using biopsy or resection samples from young patients with solid and hematologic cancers. One centimeter of tumor tissue can be segmented and implanted in multiple mice, generating 50 to 100 avatars. Coupled with the identification of gene mutations through the PIPseq program, the avatar models can then be used to determine whether the identified mutations contribute to the cancer’s development. For example, the Columbia researchers created an avatar for a toddler with aggressively metastasizing infantile fibrosarcoma, which did not have the genetic changes commonly known to be associated with this disease. As a result of sequencing efforts through the PIPSeq program, they identified and cloned a novel gene associated with the tumor’s aggressive behavior. Insight offered by genomic information and creation of an avatar model of this tumor have allowed the clinicians to choose and test an efficacious chemotherapy regimen. Researchers are also using such avatars to evaluate new drugs, with each
avatar acting as its own miniature “clinical trial” of a single drug or drug combination, including investigational agents. “We’re getting away from a one-size-fitsall approach to pediatric cancer treatment, recognizing that tumors differ from each other,” says Filemon Dela Cruz, MD, who is leading the patient-derived xenograft research with Dr. Kung. “These avatar models are already playing a critical role in allowing us to pinpoint changes driving cancer development, and to tailor therapies to target those changes.”
“Avatar” models created using a patient’s tumor tissue can be used to identify genetic mutations contributing to a cancer’s development. Above: Filemon Dela Cruz, MD (right), and veterinary technician Krysta Doust.
Genomic sequencing is not just the future of cancer medicine: We are using these technologies today to make clinical decisions for our patients.
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PIPSEQ PRECISION MEDICINE
Development of the PIPseq program was initiated by Andrew Kung, MD, PhD, with the intent of translating laboratory-based technologies to the patients cared for in the Division. The effort now brings together experts from throughout both Columbia University Medical Center and NYP/Morgan Stanley Children’s: the surgeons who remove cancerous tissue for analysis, the pathologists who prepare those biopsy samples and who perform the sequencing, laboratory scientists who use those findings to pinpoint potential new therapies, the geneticists who help interpret the results, and the oncologists and pediatric oncology nurses who administer those treatments. In January 2014, we began performing whole-genome sequencing of patients with high-risk or relapsed cancers. We are now able to offer comprehensive molecular characterization to every pediatric oncology patient we treat, with results returned to clinicians and patients within three weeks. “No other pediatric cancer program is applying precision medicine to the problem of childhood cancers the way we are,” says Maria Luisa Sulis, MD, head of hematologic malignancies in PIPseq. “We are one of the only programs prospectively sequencing cancers and using the results to make clinical decisions for our patients. Our efforts to bring the technologies of tomorrow to our patients today have extraordinary implications for defining the way we will all care for children with solid tumors and hematologic malignancies in the future.”
The PIPseq initiative is the only program prospectively sequencing pediatric cancers and using the results to make clinical decisions for patients
Julia Glade Bender, MD, is Director of the Developmental Therapeutics Program
Bringing New Therapies to Children with Cancer Access to many of the newest cancer therapies requires enrollment in earlyphase clinical trials. The Division features a very active Developmental Therapeutics Program to evaluate the safety and efficacy of new anticancer agents in children, and is one of only 21 programs in North America accredited by the National Cancer Institute and Children’s Oncology Group as a member of the Phase 1 and Pilot Consortium. Through this consortium, children have access to clinical trials in the earliest stages of testing, including drugs developed to target cancer drivers identified by genetic sequencing. In particular, these studies provide the first point of access to innovative investigational agents for children whose cancers have become refractory to standard treatments as well as other investigational therapies. We also provide patients with access to early-phase clinical trials sponsored by other national collaborative groups, such as the Dana-Farber Cancer Institute consortium and Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) consortium. Says Julia Glade Bender, MD, Director of the Developmental Therapeutics Program, “These studies allow us to offer patients access to emerging therapies when conventional therapies have been exhausted, and increase our options for matching the right drug to the right patient using our PIPseq platform.”
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A Research-Based Approach to Cancer
Our program is rooted in science, with strong laboratory and translational research initiatives and a robust portfolio of clinical trials, including pivotal early-phase studies.
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CANCER CARE THROUGH RESEARCH
Collaborating for Cures Just as it takes a village to raise a child, it takes a team to treat a child with cancer. At NYP/Morgan Stanley Children’s, subspecialists from every field within pediatrics are located onsite and are available to work as part of each child’s team. Examples include: • Pediatric neurosurgeons for children with brain and spinal cord tumors • Pediatric orthopedic surgeons for children with bone tumors
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Adolfo Ferrando, MD, PhD, and his team are exploring the molecular mechanisms underlying acute lymphoblastic leukemia — particularly leukemias that become resistant to chemotherapy
he advances that have been achieved in the management of pediatric cancers all came about as a result of research. Scientists in the Division are working in the laboratory and the clinic — and bridging the two — to accelerate the pace of such progress. For example, Adolfo Ferrando, MD, PhD, and his team are exploring the molecular mechanisms underlying the growth of acute lymphoblastic leukemia (ALL), particularly leukemias that develop resistance to chemotherapy. In a paper published in Nature Medicine, they identified a gene called NT5C2 which, when mutated, allows leukemia cells to degrade 6-mercaptopurine, an important backbone of conventional ALL therapy. “This genetic mutation may serve as a target for new therapies that could work by inactivating mutant NT5C2, restoring the sensitivity of leukemia cells to anticancer drugs,” Dr. Ferrando explains. His team has also learned that a protein called AKT1 may be involved in the resistance of leukemia cells to glucocorticoids, another critical component of ALL treatment. In a paper published in Cancer Cell, Dr. Ferrando’s team demonstrated that inhibiting AKT1 could reverse the resistance of leukemia cells to glucocorticoids. Translating these basic science discoveries to the clinic is a major priority within the Division, especially using a team-based approach partnering clinical with basic science researchers. Maria Luisa Sulis, MD, is an ideal partner for Dr. Ferrando, having previously trained in his laboratory and now serving as head of the hematologic malignancies program within the Division. Over the last ten years, Dr. Sulis has built a leukemia sample bank that has played a vital role in providing the patient samples necessary to empower basic science breakthroughs. In turn, she is now guiding the development of national clinical trials based on the resulting discoveries. “My goal is to be a champion for my patients and to help enable the bidirectional translational research that will improve the outcomes for all patients in the future,” explains Dr. Sulis.
• Pediatric liver surgeons and hepatologists from Columbia’s Center for Liver Disease and Transplantation, for patients with liver tumors • Pediatric critical care specialists for children in need of intensive care • Pediatric cardiologists, nephrologists, pulmonologists, gastroenterologists, neurologists, endocrinologists, and urologists to assist with organ toxicities that arise due to cancer or its treatment • Pediatric infectious disease specialists to help guide prevention and treatment of infectious complications • A dedicated pediatric Emergency Department for 24/7 emergency care
Maria Luisa Sulis, MD, who leads the hematologic malignancies program, has built a leukemia sample bank to provide the patient samples necessary to empower basic science breakthroughs
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CANCER CARE THROUGH RESEARCH
“Fertility has such an important impact on quality of life in our survivors that I believe no child should be treated without an explicit discussion about fertility. We must make sure we offer fertility preservation whenever possible.”
And Baby Makes Three When Erica Bullinger (Quinn), now 29, was first diagnosed with Hodgkin lymphoma in 2002, she was just 16. Becoming a mother was the furthest thing from her mind. She and her family just wanted her to get better, and she did, achieving remission after a six-month course of outpatient chemotherapy directed by her physicians at NewYork-Presbyterian/Columbia. But when her cancer returned 18 months later, more intensive measures were in order — including harder-hitting inpatient chemotherapy. That’s when her doctors started talking about fertility preservation, a topic they discussed at every visit. Freezing her eggs was not a concern for Erica or her family, however — they just wanted her to get better. After five rounds of chemotherapy as well as radiation therapy, she again achieved remission. When she reached her mid-20s and began
a relationship with Kevin Bullinger, the man she would marry, she began to seriously consider her future as a parent. Assessment of her ovarian reserve showed it was low. “They told me if I wanted to have children, I’d better start trying sooner than later,” she recalls. She and Kevin married in October 2012, when she was 27, and conceived six months later. Patrick Thomas Bullinger was born December 26, 2013. “I had an incredible experience at Columbia and have wonderful things to say about my care team,” says Erica. “They did a great job reminding me about the possible effects of the treatment on my fertility, without pushing me. It was just the wake-up call I needed!” “As a pediatric oncologist, it is our obligation to always be mindful of the future, even as we deal with the immediacy and shock of a cancer diagnosis,” says Jennifer Levine, MD, Medical Director of the Center for Survivor Wellness. Dr. Levine is a
Teamwork Moves Leukemia Research to the Clinic Dr. Ferrando and his team, including Dr. Sulis, previously identified NOTCH1 as a major driver of T-cell ALL (T-ALL). NOTCH inhibitors suppress leukemia cell growth, but cause intestinal toxicity. The Columbia investigators defined the mechanism of action of NOTCH inhibitors and identified a drug combination that maximizes their anticancer effects while minimizing intestinal side effects. Their findings
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moved the development of NOTCH inhibitors from the lab into clinical trials — which Dr. Sulis is now overseeing — to treat patients with high-risk T-ALL. “We’ve made great strides in raising the cure rate for pediatric ALL to 90 percent,” says Dr. Sulis. “The intent of our work is to help improve the outlook for the remaining 10 percent of children whose cancer is not cured using conventional therapies.”
nationally recognized leader in the field of fertility preservation in pediatric oncology, leading a task force on the subject in the Children’s Oncology Group. She adds, “Fertility has such an important impact on quality of life in our survivors that I believe no child should be treated without an explicit discussion about fertility. We must make sure we offer fertility preservation whenever possible.”
National Leadership in Hodgkin Lymphoma
Pediatric oncologist Kara Kelly, MD, is an authority on pediatric Hodgkin lymphoma and other hematologic malignancies
A Unique Center Focused on a Rare Skin Cancer Cutaneous T-cell lymphoma (CTCL) is a rare disease that primarily affects people over age 60. It is even rarer in children and is diagnosed in only 100 to 200 children each year in the United States. Nearly 90 percent of these children have early-stage disease, while a few of them are diagnosed with advanced disease that requires a more aggressive approach. Unfortunately, due to its rarity, parents find that most centers have no experience treating pediatric CTCL. NewYork-Presbyterian/Columbia has created a dedicated Pediatric Cutaneous Lymphoma Program to provide multidisciplinary care for these children. Led by Larisa Geskin, MD — a dermatologic oncologist and world leader in CTCL who was recruited to the hospital from the University of Pittsburgh in January 2014 — the care team includes pediatric dermatologists Kimberly Morel, MD, Maria Garzon, MD, and Christine Lauren, MD, pediatric oncologist Kara Kelly, MD, pathologists and dermatopathologists, and bone marrow transplantation experts. “This is such a unique cancer, and ours is the
only comprehensive multidisciplinary pediatric cutaneous lymphoma center in the world,” Dr. Geskin notes. Because CTCL cannot be cured and will last for the patient’s lifetime, the team takes a personalized and measured approach to treatment. Children with early-stage disease receive phototherapy and topical treatments, while those with systemic CTCL may initially undergo chemotherapy while waiting for a bone marrow transplant. Those who are matched with a bone marrow donor can undergo the procedure at NYP/Morgan Stanley Children’s.
Most children and adolescents with Hodgkin lymphoma can be cured, but the cure often comes with a price: a higher risk of developing long-term complications related to chemotherapy, such as second cancers and cardiovascular disease. The development of effective, less toxic therapies therefore remains a significant priority. Kara Kelly, MD, chairs the COG Hodgkin Lymphoma Committee — one of only ten disease committees within the group — and also leads large COG-sponsored national clinical trials to improve the care of patients with high-risk Hodgkin lymphoma. Because of this expertise, NYP/Morgan Stanley Children’s has become known as a major referral center for pediatric Hodgkin lymphoma. Because cancers are rare in children and adolescents, national clinical trials are necessary to learn how changes in treatment affect outcome. While some modifications are aimed at increasing the efficacy of therapy, others seek to decrease the toxicities of treatment while preserving the overall survival rate. In Hodgkin lymphoma, for example, there are clinical trials aimed at adding new targeted therapies for patients with poor prognosis disease, as well as protocols focused on decreasing the intensity of therapy for certain subgroups of patients who have achieved excellent outcomes. “While we’ve made tremendous progress in the understanding and treatment of Hodgkin lymphoma, many questions remain unanswered,” Dr. Kelly notes. “I am honored to be part of the effort to answer these questions and develop more effective, less toxic treatments for our patients today and those in the future.”
Dr. Geskin and her colleagues are also conducting research to further the understanding of pediatric CTCL. They are collaborating on an International Pediatric CTCL Registry and analyzing skin tissue, blood, and urine biomarkers that may predict a child’s prognosis. Says Dr. Geskin, “This information will help us learn which patients we need to treat aggressively and which can get by with conservative therapies.”
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Innovative Stem Cell Transplantation
We practice precision transplant medicine by tailoring treatment intensity to achieve optimal patient outcomes.
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STEM CELL TRANSPLANTATION
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he stem cell transplantation program at NYP/Morgan Stanley Children’s is one of the oldest in the nation, with expertise in treating both malignant and non-cancerous conditions. Our stem cell transplantation program is accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) and the National Marrow Donor Program (NMDP) for autologous, allogeneic, and umbilical cord blood transplantation. Our program is committed to improving outcomes for patients by decreasing the toxicities of stem cell transplantation through a two-pronged approach. First, we have pioneered novel reduced-intensity approaches to stem cell transplantation, in which we are able to give lower doses of chemotherapy with immune therapy to allow donor cell engraftment while minimizing toxicity. We have used these reduced-intensity approaches most extensively to treat patients with sickle cell disease, and have refined our approaches with over a decade of experience. We have one of the largest programs nationwide offering stem cell transplantation to patients with sickle cell disease. Over the last five years, we have achieved 100 percent event-free survival in our sickle cell patients undergoing transplant using a matched sibling donor. We are actively working to translate these exceptional outcomes to patients transplanted with alternative donor sources. Second, we offer a number of supportive therapies — including investigational therapies that are not available elsewhere — to treat or prevent the possible complications of stem cell transplantation, such as: • Photophoresis to treat acute and chronic graft-versus-host disease (GVHD), a technique in which white blood cells are removed from the patient’s bloodstream, exposed to a drug that makes the cells sensitive to light, and then treated with ultraviolet radiation to kill large numbers of the white blood cells that cause GVHD.
Quality Care — with an Eye on Cost In addition to her MD degree and expertise in pediatric stem cell transplantation, Staci Arnold has MBA and MPH degrees and performs outcomes research to demonstrate the cost-effectiveness of pediatric therapies. Stem cell transplantation can be very expensive and is often limited by insurance coverage. Although the stem cell transplantation team has always successfully advocated for patients to obtain insurance approval, the goal of Dr. Arnold’s research is to provide evidence that changes policy for all patients. She focuses not only on the cost of treatment, but also the value of effective therapies that enable children to grow up to become healthy, productive citizens. For example, Dr. Arnold recently showed that the initial cost of stem cell transplantation is quickly offset by reduced medical utilization in children cured of sickle cell disease. Importantly, their quality of life returns to normal within a year. “Families don’t need the added burden of having to struggle with financial stressors while supporting their child,” says Dr. Arnold. “If my research can help relieve that struggle by taking away the financial burden, it would be a job well done.”
• CD34-selected stem cell transplantation to prevent GVHD, where the T cells that cause GVHD are removed from the stem cell product prior to infusion into patients. • Defibrotide to treat veno-occlusive disease (VOD), a complication that may occur early in the course of transplantation, resulting in liver dysfunction due to microscopic clots. Our program has an active treatment protocol and we have defibrotide in our onsite pharmacy, allowing us to initiate therapy rapidly and achieving a dramatic reduction in mortality from this complication. • Mesenchymal stem cells (MSC), an investigational therapy to manage acute GVHD, are cells that give rise to bone, cartilage, tendons, muscles, and fat. MSC travel to areas of the body that require healing, such as tissues affected by GVHD. They are especially useful in patients with GVHD who are refractory to standard steroid therapy. • Pediatric Intensive Care Unit (PICU), which allows us to provide intensive supportive measures such as ventilator support, extracorporeal membrane oxygenation (ECMO), and left ventricular assist device (LVAD) support.
We have achieved 100 percent event-free survival in sickle cell patients undergoing stem cell transplantation from a matched sibling. 13
STEM CELL TRANSPLANTATION
We are one of only a handful of children’s hospitals offering CD34-selected stem cell transplantation, where the stem cell product is selected to enrich for cells that facilitate engraftment and deplete cells that cause graft-versus-host disease (GVHD).
Back to a Life of Dancing and Skating When Emely was 8 years old, she experienced a rapid heartbeat and excessive bruising.Her mother, Regina, brought her to NYP/Morgan Stanley Children’s — just four blocks from their apartment. She was diagnosed with severe aplastic anemia (SAA) — a disease in which the bone marrow does not make enough blood cells. While many patients newly diagnosed with SAA receive blood transfusions and/ or immunosuppressive therapy to boost blood cell counts, a bone marrow donor was quickly located for Emely. She had a stem cell transplant in August 2013.
Special Expertise for a Rare Disease
But it was no ordinary transplant. We are one of only a handful of children’s hospitals offering CD34-selected stem cell transplantation, where the stem cell product is selected to enrich for cells that facilitate engraftment and deplete cells that cause graft-versus-host disease (GVHD). “Many centers would have opted to try medical therapy first, since Emely did not have a sibling donor. But since she had a well-matched unrelated donor, and medical therapy can have toxicities, we went with transplantation as her initial therapy,” explains Monica Bhatia, MD, head of the stem cell transplantation program. “She engrafted promptly with no GVHD and was back in school much earlier than our typical BMT patients. She is now thriving!”
Our Division is known for our experience diagnosing and treating hemophagocytic lymphohistiocytosis (HLH) — a disorder in which the immune system produces too many activated T cells, natural killer cells, B cells, and histiocytes. A vital component of HLH care is its identification, since the disorder is frequently misdiagnosed. “We’ve gotten very good at diagnosing HLH early, and it has changed the course of care for some patients,” explains Prakash Satwani, MD. “Early recognition is key, because by the time many patients are diagnosed, the damage is done.” For example, some patients whose symptoms are thought to be caused by liver failure and who are referred for liver transplantation are in fact diagnosed with HLH and are properly treated with a bone marrow transplant instead. In another case, an infant referred to us for a bone marrow transplant for HLH diagnosed at another hospital turned out not to have the disorder — a finding made possible by the PIPseq program.
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Need photo of Emily
Emely, now 9, is a healthy fourth grader who enjoys dancing, skating, bike riding, and listening to music by Shakira. “The care we received at Morgan Stanley Children’s Hospital was excellent,” says her mother. “I am so thankful for all that they did for us.”
With supportive care, the boy recovered without needing a bone marrow transplant. While in infants HLH is often driven by an inherited mutation, older children with HLH may develop the disorder after infection with the Epstein-Barr virus (EBV). That was the case for Xavier, who presented to the ER at NYP/Morgan Stanley Children’s with a persistent fever and a swollen abdomen, just three days shy of his third birthday. Dr. Satwani told his mother that her son was in grave condition. After several weeks of ups and downs and an intensive pretransplant regimen aimed at controlling the EBV, he received a bone marrow transplant, followed by an infusion of his donor’s T cells that were modified to kill any remaining EBV. Today Xavier is a 4-year-old boy who lives in the Bronx and enjoys basketball, Batman, and “everything iPad,” his mother says. “From the beginning, I felt like the staff at Morgan Stanley Children’s Hospital took care of Xavier like he was their own child,” she adds. “I am so grateful to have gone there for his care and to find the doctors we needed.”
A Holistic Approach to Wellness
Integrative treatments, supportive care, and survivorship wellness are woven into each patient’s plan of care from day one.
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walk through the pediatric hematology and oncology outpatient clinic may reveal some familiar sights: Children receiving treatment. Parents speaking with nurses. Doctors checking on patients. But look closely, and you may see something else: A child getting a foot massage. A teenager receiving acupuncture. A parent learning how to use guided imagery to relax. These approaches are all part of the multifaceted Center for Comprehensive Wellness (CCW) within the Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation. Integrative therapies have been making their way into the care of people with cancer for several years. What makes this program different? Integrative treatments, supportive care, and survivorship wellness are woven into each patient’s plan of care from day one. We are also conducting the research to determine which interventions are helpful, and which patients benefit. While the PIPseq and medical programs seek to achieve more precise treatments for disease, we believe wellness care must be personalized for the child and family as a whole. “As providers, we need to have an understanding of the emotional and educational needs of our young patients at different points in their care, and provide services designed to meet those needs,” says Jennifer Levine, MD, Medical Director of the CCW. The CCW offers services such as: • Integrative therapies such as massage therapy, reflexology, acupuncture and acupressure, Reiki and therapeutic touch, exercise and yoga, aromatherapy, and mind-body techniques such as meditation and guided imagery to relieve symptoms and stress. • Nutritional counseling regarding the use of herbal and other supplements in conjunction with nutrition educators who work with children during and after treatment. • Neuropsychological assessment and psychosocial support for children and individual and group counseling for families.
• Child life specialists, who provide emotional support to patients and their families and use age-appropriate therapeutic play and education to help children understand their illness and medical procedures. • Social workers, who provide emotional support, advocacy, crisis intervention, and referrals to resources, and help coordinate a safe and secure plan when it is time to leave the hospital. • Pastoral care to provide for the religious and spiritual needs of patients and their families. • A long-term survivor program to monitor for late complications of cancer treatment and to promote survivor wellness. Reproductive endocrinologists are also available to provide fertility support, including fertility preservation, if desired. • Palliative care to improve the quality of life of patients and their families from the moment of diagnosis by relieving symptoms and helping them to understand and cope with a serious illness. 16
We are changing the way we think of “whole-person care.”
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WELLNESS FOR PATIENTS AND FAMILIES
“The doctors and all the staff blew us away. They were highly professional and open-minded, and welcomed us and our daughter. Their teamwork was just amazing.”
A Team Approach to One Little Girl’s Care As a Licensed Massage Therapist, Pamela knew every inch of her baby girl’s body. So when she felt a golf ball-sized lump in her one-year-old daughter Skylar’s abdomen, she knew something wasn’t quite right. After two months of fevers, blood tests, and ultimately an imaging exam, Pamela and her husband, Trever, learned Skylar had hepatoblastoma. They made an appointment with Darrell Yamashiro, MD, PhD, a pediatric liver cancer specialist at NYP/Morgan Stanley Children’s, traveling to Manhattan from their home in New Milford, Connecticut.
Navigating with Words It can be difficult for some children and adolescents with cancer and other serious illnesses, as well as their family members, to put their experience into words and talk about what matters most to them with each other and their healthcare
Under Dr. Yamashiro’s guidance, in April 2010, Skylar began treatment with chemotherapy to shrink the tumor to reduce the risk of complications during surgical resection. The surgery was performed by Tomoaki Kato, MD, an internationally renowned transplant surgeon, and afterward Skylar continued to receive combination chemotherapy to destroy any residual tumor cells. She completed her treatment that September. Today Skylar is a bright-eyed 6-year-old who enjoys swimming, dance class, and gymnastics and who has no evidence of disease. Although liver tumors are rare in children (they account for only one to two percent of childhood cancers), they can be very complex, and require a multidisciplinary approach for optimal management. Families of children with liver tumors who come to NYP/Morgan Stanley Children’s for their care meet with all members of the child’s healthcare team right from the beginning, and all team members collaborate regularly throughout the child’s
providers. An approach called “Narrative Medicine” — offered through the Division’s Center for Comprehensive Wellness and in association with the Columbia Program in Narrative Medicine — can make starting these conversations a little easier. The program brings patients, family members, and/or staff members together in groups of two to eight people around a piece of literature, like a poem or short story, chosen by the group’s facilitator or by one of the group members. They read it out loud, think and talk about it, and then take time to write about it. Along the way, thoughts and feelings are expressed in creative and often surprising ways. “Narrative Medicine can improve communication among all parties involved in the care of a sick child, and we know that good communication is vital to good care,” explains Chris Adrian, MD — who is not only a pediatric hematologist/oncologist,
Skylar with Darrell Yamashiro, MD, and Debra Holmberg, PNP treatment to ensure that care is delivered effectively and compassionately. Pamela was especially appreciative of the Division’s integrative medicine services, taking advantage of aromatherapy to ease her daughter’s nausea and discomfort, and chair massage for her own relaxation. “The doctors and all the staff blew us away. They were highly professional and open-minded, and welcomed us and our daughter,” she says. “Their teamwork was just amazing.”
but also an award-winning fiction writer and a graduate of Harvard Divinity School. “But it’s also a way for us to address the psychological and spiritual health of sick children and everyone involved in their care. It’s a way to ask about how people are feeling that invites a complex response.” For example, one group was asked to read a Shel Silverstein poem called “Magic,” and then asked to write about a time when they had to make their own magic. “As in the poem,” adds Dr. Adrian, “for everyone who participated, making magic in the context of a child’s illness was about a lot more than fairies and leprechauns.” Narrative Medicine is being used in the Division to allow patients, families, and staff to express themselves to each other. Says Dr. Adrian, “Narrative Medicine is another component of the care we provide that addresses the Total Child — body, mind, and spirit.”
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WELLNESS FOR PATIENTS AND FAMILIES
A Special Place for Sibs When a child is sick, the whole family hurts. Children experience anxiety and stress when a brother or sister is hospitalized. Charna’s Kids’ Club, established by the Charna Radbell Foundation’s Gift of Sunshine Fund, is a free therapeutic, restorative, and recreational program designed for children age 5 and older whose siblings are patients at NYP/Morgan Stanley Children’s. Staff members from Social Work and Child Life select activities that help them better understand their surroundings, express their feelings, and meet peers who are experiencing similar circumstances.
Palliative Care from Day One “It’s not about end of life — it’s about quality of life.” That’s what Dana Kramer, NP, tells people when she explains her role within the Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation. Unlike past definitions equating palliative care with hospice care, today’s palliative care providers work with patients and families dealing with a serious illness from the moment of diagnosis and throughout their journey with the disease.
The palliative care team speaks with parents and children about their understanding of the disease and their goals. Services range from relief of physical symptoms — such as pain, nausea, and fatigue — to nutritional guidance and emotional, social, psychological, and spiritual support. “Families in our Division have the potential for tremendous distress,” says Ms. Kramer. “Through this program, we have the potential to ease their suffering.” Our program is unique in having a palliative care nurse practitioner embedded within the Division and interacting with families throughout the course of treatment. Speaking with patients and families early in their care makes what can be a rough ride a little smoother. “This is important, because most children with cancer and nonmalignant hematologic disorders do survive,” concludes Ms. Kramer. “We want to help them get through it as comfortably as possible.”
Dana Kramer, NP, is a palliative care practitioner “embedded” within the Division who interacts with families throughout the course of treatment
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Children can enjoy arts and crafts, music, drama, special family events, group games, and other expressive activities. They can also receive help with their homework. Siblings receive support throughout the course of their family’s journey in the hospital.
Comprehensive Programs for Nonmalignant Hematologic Disorders
Being in a full-service children’s hospital allows us to provide the most comprehensive care for all of our hematology patients.
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stablished in 1959, our pediatric hematology program was one of the first in the United States. (See page 23 to learn more about our history.) Today, some 500 patients with acute and chronic hematologic disorders — from newborns to 20-year-olds — are cared for by our team, including children and adolescents with hemoglobinopathies, thalassemias, congenital and acquired anemias, bleeding disorders, clotting disorders, bone marrow failure syndromes, and vascular anomalies. State-of-the art diagnostic laboratory support from the NewYork-Presbyterian/Columbia Special Hematology Laboratory and Laboratory of Personalized Genomic Medicine enables us to diagnose rare blood disorders and render appropriate individualized treatment. In 1983, the Division became home to one of only ten National Heart, Lung and Blood Institute Comprehensive Sickle Cell Centers in the United States, a distinction we held for 20 years. The New York State Department of Health has also accredited the Division as a Hemoglobinopathy Specialty Center. We care for a large population of patients with sickle cell disease, affording us with exceptional experience managing this disease. Specialized services that we offer our patients include: • Availability of our outpatient clinic five days a week for scheduled and emergency visits, including routine preventive care, outpatient management of vaso-occlusive pain and fever, blood transfusions, and hydroxyurea therapy.
Ensuring Compliance with Sickle Cell Care Hydroxyurea is an oral medication that is beneficial for reducing the complications of sickle cell disease, but it must be taken every day to be effective. Nancy Green, MD, is studying the differences among patients in their response to this drug: the genetics of response to hydroxyurea, their adherence to therapy, and cultural and other aspects which may influence their use of the drug. She is leading the HABIT study (Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment), which is assessing an intervention using community health worker support to address existing barriers to improve the use of hydroxyurea, augmented by daily text message reminders to parents and youths. Interested in referring a patient for the study? Contact (212) 305-0494.
• Access to clinical trials, including stroke prevention protocols. • A special “Pain Pathway” in the Emergency Department and outpatient clinic to ensure the rapid evaluation and treatment of sickle cell patients with vaso-occlusive crisis, enabling these children to be relieved of their pain quickly. • A comprehensive care program to ensure that children have annual clinical, laboratory, radiologic, and psychosocial evaluations in accordance with recommended national guidelines. All of these services are facilitated on the same day, saving considerable time for families. • Access to all pediatric specialties to help treat complications of sickle cell disease. • Patients with matched stem cell donors can be cured of their sickle cell disease through our sickle cell transplant program. (See page 13 for more information.) “Being in a full-service children’s hospital allows us to provide the most comprehensive care for all of our hematology patients,” says Margaret Lee, MD, head of the pediatric hematology program. “Our program serves all of the needs of our patients in one place — not only for children with sickle cell disease, but those with any blood disorder.”
The Division’s outpatient infusion nurses deliver treatment, provide education to patients and their families, and serve as an enormous source of support. 20
Established in 1959, our pediatric hematology program was one of the first in the United States.
NONMALIGNANT HEMATOLOGIC DISORDERS
We care for a large population of patients with sickle cell disease, affording us with exceptional experience managing this disease.
Ivan (left) and Mikiel (above) were both cured of sickle cell disease, thanks to bone marrow transplantation at NYP/Morgan Stanley Children’s
Can Vitamin D Reduce Pulmonary Complications? Lung complications are leading causes of morbidity and death among patients with sickle cell disease. Infections and increased inflammation play important roles in the development of lung problems in these patients. Emerging evidence shows that vitamin D helps the immune system to fight infection and control inflammation. Through the ViDAS trial (Vitamin D for Sickle Cell Respiratory Complications), doctors from our Division — led by Margaret Lee, MD, and Gary Brittenham, MD — are collaborating with physicians from the Division of
Endocrinology, Diabetes and Metabolism and the Division of Pulmonology to see if high doses of oral vitamin D3 given once a month can reduce the rate of respiratory complications in children and adolescents with sickle cell disease. “Many patients with sickle cell disease are already deficient in vitamin D,” notes Dr. Lee. “If the results are positive, vitamin D could represent a simple, inexpensive, noninvasive treatment for preventing respiratory complications of sickle cell disease.” Findings from the study are expected in summer 2015.
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NONMALIGNANT HEMATOLOGIC DISORDERS
“The entire team cared for me, my child, and my family and lent a helping hand when needed. If you need to go through this experience, Morgan Stanley Children’s Hospital is where you should go through it.”
A Brother’s Gift Bryce was diagnosed with sickle cell disease when he was 3 years old. His parents, Tiffany and Alex, knew about the potential complications of sickle cell disease — Tiffany was particularly worried about the risk of stroke — and were referred to NYP/Morgan Stanley Children’s by their pediatrician. Bryce had already been hospitalized once, at age 1, to receive a blood transfusion. They learned that a bone marrow transplant offered the only chance of a cure. His baby brother, Brandon, born December 2010, was a perfect match and served as Bryce’s bone marrow donor. Bryce was in the hospital for three and a half months to receive his transplant
in the spring of 2012. “The exemplary care we received was far more than I expected,” says Tiffany. “The entire team cared for me, my child, and my family and lent a helping hand when needed. If you need to go through this experience, Morgan Stanley Children’s Hospital is where you should go through it.” Today Bryce is a vibrant young boy who enjoys taking swim lessons, which he could not do when he had sickle cell, due to the risk of pain crisis. Tiffany, an assistant principal for a public middle school in the Bronx, likes to speak about her experiences to let other parents of children with sickle cell disease know about their options. “In the community where I work, I see children with this disease who are not being treated, and
I tell them my story. The team here is very good at helping parents understand the options available to them,” she says. “Your child does not have to live with this. There’s a light at the end of the tunnel.”
A Helping Hand — and Heart — for Kids with Sickle Cell While stem cell transplantation can cure some children with sickle cell disease, many more must live with the disease for life. Maureen Licursi, NP, says it is her job to help patients and their families learn how to understand, accept, and grow with the disease. Sickle cell crises can bring patients to the hospital in severe pain. However, there are therapies they can take to manage their discomfort, ranging from anti-inflammatory agents to opioids to warm packs. Ms. Licursi helps each family create a customized “toolbox” containing the treatments that work to manage their child’s pain. “My goal is to keep children at their family dinner tables each evening and in their own beds every night, not in the hospital,” she says. “Moreover, pain crises can be exacerbated
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by ‘haunted memories’ of prior crises, so we start managing them as early as we can to prevent those scars.” Ms. Licursi — who has worked in the outpatient hematology clinic at NYP/Morgan Stanley Children’s for 12 years — not only discusses treatment options with patients (such as transplant as a curative therapy and hydroxyurea as a diseasemodifying modality), but also talks about how to live with sickle cell disease. At each visit, she looks for ways to connect with children and their parents and whatever events may be going on in their lives. When a child is about 15, she starts to discuss the transition to adult care, which happens when patients reach age 20, so they can learn how to advocate for their own health.
“Sickle cell is a condition that affects families,” she concludes, “Through my job, I can really influence lives — not just for the children, but for their families.”
Dr. James A. Wolff
A LEGACY OF LEADERSHIP
The Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation at Columbia University Medical Center was one of the first in the country to provide dedicated hematology and oncology care for pediatric patients. It was created in 1959 at Babies Hospital — the forerunner of NewYork-Presbyterian/ Morgan Stanley Children’s Hospital — under the leadership of the late James A. Wolff, MD, a pioneer in pediatric hematology who was on the front lines of cancer research during the earliest days of chemotherapy.
Dr. Wolff’s interest in hematology stemmed from his experience on the transfusion service of a field hospital in the European Theatre during World War II. As a Resident at Boston Children’s Hospital in the mid-1940s, Dr. Wolff worked with Drs. Sidney Farber and Louis Diamond, and in 1948 they reported for the first time the effectiveness of the drug aminopterin against childhood leukemia. This milestone in oncology heralded the beginning of extraordinary progress in the battle against pediatric cancers, in which acute leukemia turned from a once uniformly fatal disease into one that is now curable in more than 85 percent of affected children.
With Dr. Wolff’s appointment as Director of Pediatric Hematology at Babies Hospital in 1959, the early research activities of the Division focused on the treatment of erythroblastosis fetalis and children with thalassemia major. Babies Hospital established a combined hematology and oncology clinic, and was one of the first to use a multidisciplinary approach to treating childhood cancers. Dr. Wolff was one of the first members of the Children’s Cancer Study Group, which became today’s Children’s Oncology Group — the research consortium whose efforts would ultimately save the lives of thousands of children with cancer. “Jim Wolff was present at a time when hematology was transformed from an observational practice into today’s scientific disciplines of hematology and oncology,” explains Gary Brittenham, MD, the James A. Wolff Professor of Pediatrics. “He was ‘the man’ to see for hematological disorders. He inspired devotion among those he led.” “He was a remarkable man and a very compassionate physician, with a wide array of interests,” adds Michael Weiner, MD, Vice Chair of the
Department of Pediatrics. In 1979, Dr. Wolff was succeeded as Director by Sergio Piomelli, MD, who continued the strong research emphasis of the program. When Dr. Piomelli retired, the Division of Pediatric Hematology merged with the Division of Blood and Marrow Transplantation, with an independent and distinct Division of Pediatric Oncology. In 2007, a separate Division of Pediatric Hematology was established. In 2012, the separate Divisions were re-united as the Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation, under the direction of Andrew Kung, MD, PhD. Dr. Wolff died in December 2012, having left an indelible mark on the field of pediatric hematology/oncology, the hospital, and the Division.
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Hope & Heroes
At Columbia University Medical Center, there is a unique community of donors dedicated exclusively to the cause of fighting children’s cancer and blood disorders through their support of a nonprofit organization called Hope & Heroes. Founded by Michael Weiner, MD, in 1997 and given 501(c)(3) status in 2002, Hope & Heroes was an idea born of necessity. With an ambitious vision for a state-of-the-art research and treatment center, Dr. Weiner knew that private funding would be essential to the success of the enterprise. Today, the impact of philanthropy on the work of the Division is striking, with a full 40 percent of the budget of the Division of Pediatric Hematology, Oncology and Stem Cell Transplantation provided by Hope & Heroes. Support from Hope & Heroes is used to underwrite the Division’s innovative approach to clinical care — one that emphasizes caring for the child and the family as a whole — and makes possible services that are not reimbursed by insurance, such as integrative therapies, wellness programs, psychosocial support, educational support, and
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survivorship programs. As pediatric oncology nurse and long-time Hope & Heroes supporter Shira Kapito Arcus states, “There’s a sense of vulnerability among children with cancer and their families, and we become so close with them as we support them on this journey.”
HOW DONORS HELP IN THE FIGHT AGAINST CHILDREN’S CANCER AND BLOOD DISORDERS Hope & Heroes also underwrites cutting-edge medical research in the Division with the potential to dramatically improve the future of patients with cancer and blood disorders. Although tremendous progress has been made in the treatment of many forms of childhood cancer, the National Cancer Institute dedicates less than five percent of its annual budget to pediatric oncology research. Funding from Hope & Heroes has allowed the Division to recruit top translational and clinical researchers — including the Division’s Chief, Andrew Kung, MD, PhD — and to sustain the breakthrough research that will continue to improve outcomes for young patients with cancer and blood disorders. Hope & Heroes raises money through direct appeals and by hosting a number of events each year, including a luncheon in April, the annual Hope & Heroes Walk each May, a golf outing in June, and a gala dinner celebration in November. The breadth and caliber of patient care made possible by supporters of Hope & Heroes is remarkable. As Dr. Weiner says,“People respect and want to participate in improving the lives of children with cancer and blood disorders. Hope & Heroes enables us to provide resources that make the lives of children and their families better, and to continue to improve outcomes.” For more information about Hope & Heroes or to get involved, please call (212) 305-1420 or email contact@hopeandheroes.org.
About NewYork-Presbyterian/Morgan Stanley Children’s Hospital
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Contact Us To learn more about the programs and services of the Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation at NewYork-Presbyterian/ Morgan Stanley Children’s Hospital, please call (212) 305-9770.
NewYork-Presbyterian/Morgan Stanley Children’s Hospital at Columbia University Medical Center, located in New York City, offers the best available care in every area of pediatrics — including the most complex neonatal and critical care, and all areas of pediatric subspecialties — in a familyfriendly and technologically advanced setting. Building a reputation for more than a century as one of the nation’s premier children’s hospitals, NYP/Morgan Stanley Children’s is affiliated with the Department of Pediatrics at Columbia University College of Physicians and Surgeons. In addition, each of our physicians is a member of the highly regarded ColumbiaDoctors faculty practice. We are Manhattan’s only hospital dedicated solely to the care of children and one of the largest providers of children’s health services in the Tri-State area, with a longstanding commitment to our community. We are also a major international referral center, meeting the special needs of children from infancy through adolescence worldwide. Whatever a child’s medical needs, we have the specialists and subspecialists on our team to meet them. NewYork-Presbyterian is the #1 hospital in the New York metropolitan area, according to U.S. News & World Report, and is consistently named to the magazine’s Honor Roll of best hospitals in the nation. For more information, visit NYP.org/KIDS/MorganStanley. NewYork-Presbyterian/Morgan Stanley Children’s Hospital 3959 Broadway (at 165th Street) New York, NY 10032
NewYork-Presbyterian/ Morgan Stanley Children’s Hospital 3959 Broadway (at 165th Street) New York, NY 10032 (212) 305-9770 NYP.org/KIDS/MorganStanley