Newsletter - Thalassaemia Prevention Porgramme by Thalassaemia International Federation (TIF)

Issue Aug 2016

FATIMID FOUNDATION Symbol of Hope

We try to achieve managerial and technical excellence in the field of blood banking and haematological services. Fatimid Foundation engage professional manpower in the field of blood banking and haematological services.

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We provide first class quality blood banking and haematological services safely to underprivileged patients and the community at large, we ensure the use of FDA/CE approved high quality materials and equipment for the treatment of thalassemia haemophilia and other genetic blood disorders.

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COOâ&#x20AC;&#x2122;s MESSAGE

Fatimid Foundation is a not-for-profit organization. CDP has supported and funded Fatimid foundation for three Projects. We completed our first project of Thalassaemia Prevention and Awareness programme with the help of CDP in 2009 and screened 6006 members of Extended families of diagnosed thalassaemia major patients from Karachi and Hyderabad. Second Project began in 2011 and it was completed in 2012. We screened 522 members of extended families of diagnosed thalassaemia major patients from Tando Allahyar (Rashidabad). In the third Project, our target is to screen 9000 extended members of thalassaemia major patients from Karachi, Badin, Thatta, Tando Mohammad Khan and Tharparkar. We have now screened 9000+ members.

Lt. Gen. (R) Moinuddin Haider Chairman

The Projects:

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We have throughout our history taken bold steps to fulfill our mission to make a difference for our underprivileged patients and blood donors who depend on us. This would not have been possible without the generous and continuous support of its donors which includes individuals, corporate sector, local and foreign Governments.

Fatimid Foundation have begun to develop a multi disciplinary research consortium and enhance donor and patient care. We collaborated with reputable hospitals and overseas bodies to bring about individual strength and expertise to fully round out our strategic abilities.

In June 2009 with collaboration of Community Development Programme (CDP), Planning and Development Department Government of Sindh, Fatimid Foundation began thalassaemia prevention programme. The objective was to screen for thalassaemia minor in extended family of diagnosed thalassaemia major patients and prevent the birth of thalassaemia major patients through prenatal diagnosis (Chorionic villus sampling). By doing this we are able to detect carriers, avoid marriages between two minor individuals, avoid birth of Thalassaemia Major foetus and discourage consanguineous marriages.

This has been a year of coming together, of cohesion for Fatimid Foundation. We have begun to change the structure of our operations to concentrate on expertise and capacity to prepare for growth.

Fatimid Foundation with Collaboration of Community Development Programme, Planning & Development Department, Government of Sindh By Nazneen Zaidi, Consultant Gynecologist & Project Director

Chairmanâ&#x20AC;&#x2122;s Message

THALASSAEMIA PREVENTION PROGRAMME

Impact: The number of marriages between thalassaemia minors decreased by approximately 60 - 70%. The number of thalassaemia major births to thalassemia minor couples decreased by approximately 30 - 40% through prenatal diagnosis (CVS). There was increased awareness regarding thalassaemia and its prevention among general population.

We provide in-house and outside training and development to staff in all functional areas & ensure complete transparency, good governance and management practices as a non-profit organization.

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SUCCESS STORY

By Dr Shabneez Hussain, Consultant Haematologist, Fatimid Foundation

“Cardiac iron overload is reversible and every patient has a chance to be saved”

Miss Fiza haroon rasheed (T 1883) is a 13 year old female, diagnosed case of thalassemia major who presented to the haematology clinic with high grade fever, shortness of breath and palpitation. On examination she had splenomegaly (8cms), pedal edema and bilateral crackles. She was poorly chelated (desferrixamine 4 days a month). Due to the pancytopenia resulting from splenomegaly, she was unable to take deferripirone. Her serum ferritin was was 7800 ng/ml. She was diagnosed as having heart failure. Her T2 MRI was 2.6 ms (severe cardiac iron overload). She had diastolic and systolic dysfunction on echocardiography with an ejection fraction of 57%. She also had atrial tachycardia on ambulatory ECG (Holter). She was admitted at The Aga Khan University Hospital and begun on an emergency management with intensive iron chelation. Twenty four hour subcutaneuous /intravenous desferroxamine infusion and deferrasirox were advised. She was treated for heart failure She continued with intensive chelation for the duration of 1 month. Following that the duration of desferrioxamine was reduced to 8 to 12 hours and deferrasirox was continued. A pre transfusion haemoglobin target of 9 gm/dl was set. Heart failure resolved after 1 week of intensive management. After 6 months, echocardiography was repeated it showed resolution of systolic and diastolic dysfunction with improved. Serum ferritin was 4726 ng/ml. With proper transfusion regime, her spleen size reduced to 6 cms. The management of heart failure in a thalassemia major is completely different from that of an adult. Fiza was initially admitted at a nearby hospital and was treated according to the protocol of adult heart failure patient. She was near death but with a motivated mother and her own determination we were able to save her, Dr. Babar Hassan of Aga Khan University Pediatric Cardiologist, played a very important role in her cardiac management. Currently, she is well and able to perform her daily activities. Once the T2 MRI is above 8 ms, she will be switched to a single oral iron chelating agent, preferably deferrasirox.

CHELATION THERAPY FOR IRON OVERLOAD

By Dr Shabneez Hussain, Consultant Haematologist, Fatimid Foundation

Thalassemia major is a major prevalent disease in a country like Pakistan where illiteracy, poverty and disease go hand in hand. There are two key aspects that need to be targeted when managing thalassemia major patients; optimum transfusion and optimum iron chelation therapy. The chelation therapy described here is meant to be prescribed depending on the socioeconomic status, adverse effects of chelating agents and patient preference.

Chelation is begun when either one of the following has occurred: 1. Ferritin >1000 ng/ml 2. More than 10 transfusions The choice of chelating agents depends on the following: 1. Desferrioxamine : All ages 2. Deferasirox: >2 years 3. Deferiprone: >6 years Affording Patients: Recommended therapy: Ferritin >2500 • Desferrioxamine 40mg/kg 5 days a week with Deferiprone 100mg/kg daily Or • Desferrioxamine 40mg/kg 5 days a week with Deferasirox 30 to 40 mg per kg Ferritin <2500 • Deferiprone 75 to 100 mg/kg daily with Desferrioxamine 35 mg/kg or Defera sirox 30 mg/kg 2 to 5 days per week to maintain ferritin at 500 • If Deferiprone contraindicated then Deferasirox 30 mg/kg daily with Desferriox amine 25 to 35 mg/kg 2 to 5 days a week. If T2 MRI Cardiac, echocardiography and ECG Holter is available: T2* 8 -20 ms, ECHO and ECG Normal: • Desferrioxamine 40 mg/kg/d for 12 hours SC infusion daily with vitamin C or • Deferiprone 75 to 100 mg/kg/d daily or • Deferasirox 40 mg/kg/d daily T2* <8ms and LVEF >56% and ECG Normal: • Desferrioxamine 50 mg/kg/d for 12 hours SC daily and deferiprone100 mg/kg/d or Deferasirox 40 mg/kg/d daily T2* <8ms, LVEF <56% and / or ECG reveals arrhythmia: • Desferrioxamine 50 mg/kg/d 24 hour IV infusion / SC for 15 days (if no arrhyth mia) and 30 days (if arrhythmia or heart failure) and deferiprone 100 mg/kg/d daily • Once that is complete then desferrioxamine 50 mg/kg/d 12 hour SC and deferiprone 100 mg/kg/d daily for 1 year then repeat MRI/ECG/ECHO. • If Deferiprone is contraindicated then can use Deferasirox 40 mg/kg daily. Alternate Chelation Therapy for non affording patients or those intolerant to desferioxamine An alternate form of chelation therapy has recently shown an equivalent result when compared to Desferrioxamine and Deferiprone/Deferasirox. This is the combination of both oral chelating agents; deferasirox and deferipirone. Although not yet published in “thalassemia international federation” guidelines, it has shown similar efficacy if not superior due to its far better compliance. FAQs on chelation therapy: • Give Deferasirox after meals in orange juice. Do not use metal utensils. Keep afixed timing of ingestion. Initially begin with 20 mg/kg for 1 month. Can increase upto 40 mg/kg. Discontinue if creatinine above normal range, SGPT and SGOT more than 4 to 5 times above baseline. • Deferiprone should be taken after meals to decrease the chance of gastric intol erance. Initially begin with 50 mg/ kg for 6 to 8 weeks with CBC every week for 3 weeks. Discontinue if ANC < 1.5 and platelet <100 X109/l. • Target ferritin should be between 500 to1000 for thalassemia major and <400 for thalassemia intermedia.

WHAT IS T2* MRI! By Najveen Alvi (Research Associate,Department of Paediatrics, Aga Khan University) and Dr. Babar S. Hasan. (Associate Professor Department of Paediatrics and Child Health Aga Khan University.)

• Do not use desferrioxamine 40mg/kg/day or higher in children who are <15years of age. In adults a dose of up to 50mg/kg/day should be used if there is concern about cardiac iron loading. • Vitamin C (prescribed with desferrioxamine) should not be given in severe cardi ac iron overload. • T2 MRI (Only being performed at AKUH) should be performed on all patients once they reach 10 years of age. • If baseline T2 MRI is > 20ms (normal) then it can be repeated every 2 to 3 years provided the patient is on optimum chelation. Acknowledgements: Dr Farrukh Shah, Haematologist, Whittington Hospital, UK Repeated regular blood transfusion is the basic therapy for patients with Thalassaemia. It is proven to improve oxygen-carrying capacity, and different parameters of growth, development, and overall well-being of the patients. However, regular transfusions are known to lead to an excess of iron accumulation within the body, including vital organs like the liver and heart. The excess iron within the heart can adversely affect the function of the heart. This is considered the most important basis of survival in Thalassaemia. Different clinical tests including ferritin levels look at the overall iron levels of the body. However, these tests do not directly measure the iron within the heart. Cardiac (heart) T2* MRI is the only direct and valid measure of exact heart iron levels. It directly measures and quantifies the level of iron within the heart. T2* MRI values above 20 ms are considered to be normal. Why should I get it done and what does the result show? Cardiac T2* MRI allows clinicians to directly measure the iron level present within the heart. Values above 20 ms are considered to be normal. Values below 20 ms represent increased presence of iron in the heart and require special attention. Patients with values between 15-20ms are considered to have mild iron excess and those between 10-15ms have moderate iron excess. Values below 10 ms represent severe iron load in the heart and require urgent management. By quantifying cardiac iron, T2* MRI allows for clinicians to take adequate action as needed to protect the heart before the function declines. It is known that T2* values predict the likely course of heart function up to one year ahead of time. Therefore cardiac T2* values allow for better targeted chelation as well as heart specific therapies.

The patient or anyone accompanying the patient in the MRI room should not be carrying metallic objects including pens, pocketknives, jewelry, watches, credit cards, and hearing aids and removable dental work, pins, metal zippers, and similar metallic items. When should it be done and how often should I get it done? Since the imaging requires breath-hold, some of our younger patients may not be able to perform the test properly. Therefore, we suggest that children younger than 7 years of age may not be suitable to get the MRI. This is an ideal test to offer all our patients 7 years old and above. Once done, the subsequent follow up T2* MRI is to be determined by the test results. Generally, patients with normal values are recommended to repeat this MRI every year. For patients demonstrating high iron levels in the heart (T2* less than 10 ms) the test should be repeated earlier as per the suggestion of the treating cardiologist and hematologist.

VISIT OF WORLD HEALTH ORGANIZATION (W.H.O) AND SINDH BLOOD TRANSFUSION PROGRAMME (S.B.T.P)DELEGATES By Dr Shahida Baloch, Director Quality Assurance On 11th February 2016, Dr. Yatmgeta Abdella, Head of Blood Safety Unit from W.H.O and Mr. Usman Waheed, SBTP coordinator and consultant, visited Fatimid Foundation. Mr. Rumi Dossal (Chief Operating Officer) and Dr. Shabneez Hussain (Consultant Haematologist) gave a detailed update on the different ongoing projects and patient management protocols of Fatimid Foundation.

How is it done? Cardiac T2* MRI is an MRI, an imaging method that uses powerful magnets and radio waves to create pictures of the heart. It does not use radiation (x-rays). The patient will be asked to lie on the MRI table. Electrocardiogram (ECG) leads (small sticky patches) will be placed on the chest to help the machine synchronize the images taken with the heartbeat. The patient will be given breathing instructions and will be asked to hold the breath numerous times during the examination.This is very crucial and care must be taken to ensure adequate and complete breath-hold. This is needed to acquire clear accurate images of the heart. Because the MRI contains strong magnets, metal objects are not allowed into the room with the MRI scanner. A patient with the following may not be eligible for a T2* MRI • • •

Metallic artificial heart valves, vascular stents or brain aneurysm clips. Heart defibrillator or pacemaker Recently placed artificial joints or surgical pins and wires.

Mr.Rumi Dossal briefed that Fatimid Foundation was extending quality healthcare facilities free of cost to the non affording Thalassaemia and Haemophilia patients of the country. Dr. Abdella visited all the sections of Fatimid Foundation that included Thalassaemia dept., haemophilia department, medical record room, laboratory and apheresis room. He appreciated the enthusiasm and dedication of the staff of Fatimid and extended his best wishes for further developments.

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VISIT OF THALASSAEMIA INTERNATIONAL FEDERATION (TIF) DELEGATION TO FATIMID FOUNDATION Dr Shahida Baloch (Director Quality Assurance) and Dr Shabneez Hussain (Consultant Haematologist)

Thalassaemia Federation of Pakistan 10th Annual Congress was held on November 27th -29th 2015 in Karachi. The Thalassemia international federation (TIF) delegation, comprising of Dr Michael Angastiniotis, Mr Stavros Melides and Dr Farrukh Shah, also attended this conference.

Mr. Melides is a father of a thalassaemic child side and besides being associated with TIF, he is also working with the Cure2Children organization of UK. Dr. Farrukh Shah is a trained haematologist from University College of London, UK. She was appointed as a Consultant Haematologist at the Whittington Hospital in 2004. She became Fellow of The Royal College of Pathologists in 2008. Dr Shah has a specialist interest in the management of transfusional iron overload and haemoglobinopathies. She is actively involved in clinical research in iron chelation and is a local and national investigator for a number of clinical trials. She is a medical adviser to the UK Thalassaemia Society. She speaks a number of languages, which has enabled her to communicate directly with some of the communities from India and Pakistan.

Dr. Michael Angastiniotis a renowned pediatrician having a special interest in thalassaemia management and prevention. He is a member of the Board of Directors and a close collaborator with the thalassaemia lab at the Cyprus Institute of Neurology and Genetics. He is a medical advisor to the Thalassaemia International Federation.

This delegation visited Fatimid Foundation on 28th November 2015. Mr. Rumi Dossal welcomed the honorable guests. They visited various departments and gave valuable advice to doctors regarding various treatments protocols. Dr. Shabneez Hussain (haematologist, Fatimid Foundation) and Dr. Zia ur Rehman (Medical Director), Dr Ejaz Brohi briefed about patientâ&#x20AC;&#x2122;s treatment. Dr. Nazneen Zaidi (Gynecologist) discussed the thalassaemia prevention projects. The delegation appreciated the efforts of Fatimidâ&#x20AC;&#x2122;s Staff in thalassemia management and prevention. They helped us set up milestones for the future.

Editor: Dr. Shabneez Hussain Co Editor: Dr. Shahida Baloch

AJM Pharma in collaboration with Fatimid Foundation to treat Thalassaemia patients.