Newsletter - June 2015
Sibling Transplants for Thalassemia: Family Banks Fulfill a Public Health Need
Frances Verter, PhD
The inaugural Cord Blood Industry Report from the Parent's Guide to Cord Blood Foundation reveals that sibling transplants for thalassemia are becoming the dominant allogeneic therapy with cord blood from family banks. An allogeneic therapy is any therapy where the cord blood is used for a person other than the baby from which it originated. A pie chart of the cumulative allogeneic therapies through the end of 2013 is shown at left.
Our Cord Blood Inustry Report also finds that most of the growth in sibling transplants for thalassemia is occurring in Asia. Considering the prevalence of thalassemia among populations in Asian nations, coupled with the growth of family cord blood banks in that region, this trend is expected to continue.
Parent's Guide to Cord Blood (PGCB) Foundation is a registered 501(c)(3) charity based in the United States. For over a decade, the PGCB Foundation has been taking confidential surveys of the inventory of cord blood stored in family banks and the number of cord blood therapies released from family banks. These surveys are confidential in the sense that the identity of individual banks and individual patients are not revealed, the data are only reported in anonymous and/or aggregate statistics.
Starting this year, the survey is being published as a subscriber service to a secure website CordBloodIndustryReport.org. A preview of the report was published in Ballen, Verter & Kurtzberg 2015, and a different preview is provided here.
Some cord blood banking statistics across the decade from 2003 to 2013:
|Year||Cumulative 2003||Cumulative 2013|
|Worldwide therapies released from family banks||102||1015|
|Global family therapy division allogeneic/autologous||93% / 7%||52% / 48%|
|% Family allogeneic therapies for blood cancers||53%||37%|
|% Family allogeneic therapies for hemoglobinopathies||26%||39%|
|Ratio of therapies from Public/Family banks||30||35|
The figure below shows the growth of allogeneic therapies from family cord blood banks versus time. In this graph the therapies are divided by the diagnosis categories: thalassemia, blood cancer, sickle cell, rare disease, brain injury, and miscellaneous. Over the four years concluding in 2013, thalassemia transplants have equaled or exceeded transplants for blood cancers. In 2013 the transplants for thalassemia alone exceeded all other allogeneic therapies combined.
About 15 million people worldwide were sick from thalassemia in 2013, making it a public health issue in those countries with many carriers. Throughout Asia many families carry one of the genetic mutations for thalassemia. In Northern Thailand and Laos, the prevalence of alpha-thalassemia is 40%, while beta-thalassemia varies between 1-9%. The hemoglobin E (HbE) mutation also occurs in 30-40% of this population and reaches 60% prevalence in certain areas of North East India. These abnormal globin genes, present in different combinations, lead to over 60 different thalassemia syndromes. (Manipalviratn et al. Parent's Guide to Cord Blood Nov. 2014 newsletter)
The PGCB Foundation recently contributed to a book titled Stem Cell Banking & Transplantation, in which 49 of the 55 contributors are based in India (editors Dhot, Brig. Dr. PS, Parikh, PM, & Mishra, Col. D. 2015). Current estimates suggest that about 10,000 children are born in India each year with Thalassaemia. (Brig. Dr. Dhot, p.244) "The burden of transfusion and chelation in children with thalassaemia in India is huge and the majority of patients in our country face early death in their teenage years due to cardiac or liver haemosiderosis... With the advent of preimplantation genetic diagnosis and savior siblings to help children with thalassaemia, we do foresee a greater role for the use of cord blood stem cells in this group of children in the coming years." (Dr. Revathi Raj et al., p.235)
Meanwhile, family cord blood banking has become a routine practice at the private hospitals used by relatively affluent parents throughout Asian nations. China launched a network of state-run public/family (known as the hybrid business model) cord blood banks since 2003, and privately-owned family banks took off in India since 2004. India has the world's highest number of annual births, at 24.6 million in 2014, and China is next at 16.5 million ( CIA World Factbook on-line).
If the Thalassemia case reports from family banks over the past ten years of the PGCB confidential survey are separated according to whether they were transplanted in Asia or elsewhere, it is clear that the Asian banks are responsible for the rise in thalassemia transplants.
In recent years the majority of the thalassemia transplants from family banks in the PGCB Foundation survey took place in China and India. This is illustrated in the pie chart to the left, which shows thalassemia transplants for 2013 only, divided by nationality. As of 2014, the PGCB Foundation survey found that 34.6% of the world’s family cord blood inventory is in the nations of Asia & Oceania.
In summary, it is clear that family and hybrid cord blood banks are becoming an important health resource for families in Asia that carry a thalassemia trait and especially those families who already have a child that is sick with thalassemia. These communities need to also build their inventory of public cord blood donations so that more families can access cord blood transplants as a curative therapy for thalassemia.
This study demonstrates that family cord blood banks can provide an important public health service in parts of the world where the public banking infrastructure is weak or non-existent.
AMTF - Helping Blood Disorders
Asim Qidwai, MD
AMTF stands for Afzaal Memorial Thalassemia Foundation. AMTF - Helping Blood Disorders is a charitable foundation that was launched in November 2003 by Dr. Asim Qidwai along with a group of doctors, philanthropists, social workers, and parents of children suffering with blood disorders, sharing a vision of developing a center in Pakistan with a multidisciplinary approach for the treatment of children with blood disorders.
The mission of AMTF - Helping Blood Disorders is to develop an institute for pediatric hematology with a well-built research facility in Pakistan where quality clinical care will be given regardless of ability to pay. AMTF's vision is to deliver the utmost level of compassionate care and to raise awareness about pediatric blood disorders.
The thalassemia parents had a strong belief that following the leadership of Dr. Qidwai the project would be accomplished with pride and serving the prestige of those underprivileged by providing care and cure services free of cost under one umbrella named as AMTF.
Initially the project consisted of a two room clinic, 3 employees, and a day care unit with a capacity for 5 to 7 patients. Over a period of time this project has progressed and now by 2015 has become a three floor hospital that houses Pakistan's first structured program for Haemoglobinopathies (please refer to our website to read the journey).
At present AMTF has more than 120 employees working in this center and more than 900 registered children with different kinds of blood disorders along with a day care capacity of more than 15 patients each shift. We have a 24 hrs well-equipped laboratory with the following departments: microbiology, biochemistry, hematology, blood bank, cytology.
In Pakistan, AMTF is first of its kind organization that is serving the victims of any such genetic/hereditary chronic blood disorders like thalassemia and other haemoglobinopathies with an approach of multidisciplinary care team and support facilities. The team comprises of Pediatrics, Cardiology, Hematology, Haemoglobinopathies, Endocrinology, Psychology, Genetic Counseling, Neurology and General Pediatrics, all working altogether for thalassemia patients as well as patients with other hematological disorders. AMTF has a dedicated nursing team working under the supervision of an expert nursing educationist and has also developed pharmacy services managed by qualified pharmacists.
AMTF has a dedicated pharmacy team that introduced a pharmacovigilance program with the concept of an iron chelating agent toxicity monitoring clinic. The clinic started successfully with a pharmacist, nurse practioner, and physician providing real time data collection.
In 2012 Dr. Asim Qidwai took the challenge for the development of Pakistan's first intensive care unit for patients with blood disorders and childhood cancers and later on this project became functional in 2013 with collaboration from the Government of Japan as Pakistan's first thalassemia intensive care unit. Also as part of this collaboration we started Pakistan's first pediatric cardiology clinic for thalassemia patients that has facilities for ECHO, ECG and free consultation for thalassemia patients from cardiologists.
AMTF started Pakistan's first psychology clinic for thalassemia parents and patients to rule out anxiety, depression and other problems. The clinic also established availability of psychology therapies to improve the quality of life for thalassemia patients and their parents.
All medications, in-patient services, diagnostic services and intensive care services are available to our registered patients 24 hrs and we also provide services to patients of other centers depending on the availability of our capacity and capability.
In general, AMTF is catering mostly to thalassemia major patients, with a handful of cases of sickle cell disease, platelet disorders, and aplastic anemia. We provide all of these services free of cost with pride.
Dr. Asim Qidwai has set AMTF's next goal as the development of a state of the art center for benign hematology that can provide transplant services along with all of the above care facilities. This task will be the next generation of AMTF.
We would like to thank all of our supporters for providing unconditional support to AMTF. In the future we expect more support and collaboration at the national and international level so that our team will get exposure to international standards and at the same time we can share our experience globally.
The AMTF concept "OUR PATIENTS ARE OUR GUESTS" has been a guiding element in our quest to improve the standard of care and to respect children suffering with blood disorders. We request each soul from every walk of life to come forward and take part in the development of this noble cause. We are looking forward to the participation of the community at large. For more information please contact us at email@example.com
Dr. Asim Qidwai has a special interest in Haemoglobinopathies and childhood cancers. He is a founder of AMTF Helping Blood Disorders program in Karachi, Pakistan. Dr. Asim Qidwai did his medical school in 1995 followed by residency in pediatric medicine, a fellowship in pediatric hematology and oncology, and a Masters in Haemoglobinopathies from University College London. In 2014 Dr. Qidwai was awarded with a Gold Medal for his services in the establishment of Pakistan's first intensive care unit for children suffering from blood diseases and cancers.