Dr. Prantar Chakrabarti

Shining Light on Clinicians Serving the Hereditary Blood Disorders Community Introducing Dr. Prantar Chakrabarti MD, DBN, DM, a renowned Clinical hematologist with a specialty in Thalassemia, Hemoglobinopathies, and Bleeding disorders like hemophilia A, hemophilia B, and Von Willebrand disease.

GANSID: What hereditary blood disorder/s do you treat and what leads you to that specialty area? Dr. Prantar: The hereditary blood disorders that I usually treat are thalassemia, hemoglobinopathies, and bleeding disorders like hemophilia A, hemophilia B, and Von Willebrand disease. I also take care of inherited platelet disorders like Glanzmann, Thrombasthenia, and Bernard-Soulier syndrome. Some of the primary immune deficiency syndromes are also referred to me and I take care of them in collaboration with pediatricians and immunologists. After specializing in internal medicine, I joined the Institute of Hematology at Medical College Kolkata in 2002 and hence my journey in this specialty started. Later, I did my postdoctoral fellowship training i.e. (DM in Clinical Hematology ) from All India Institute of Medical Sciences, New Delhi which then officially certified me as a clinical hematologist. I started teaching and ultimately became the professor and head of the department of Hematology at NRS Medical College Kolkata. Realizing the limitations of becoming a social activist while in a government job, I quit my job in 2019 and am trying to develop a comprehensive, accessible rural healthcare model empowering rural folk with the help of information technology and artificial intelligence. GANSID: Can you share a major challenge you have encountered in your practice? Dr. Prantar: The 2 major challenges I've encountered in my practice are the sheer number of patients who used to come to a single OP clinic at NRS Medical College, Kolkata, and the poor mental health status of the patients and their family members. The average number of patients who were attending the thalassemia clinic was close to 250 patients on a single day. According to the rules of the government, we could not schedule any appointments. So, it was a walk-in clinic where any patient could walk in for a consultation. Realizing that we could not do justice to any of the patients as the average consultation time would be less than 2 minutes, we sat together and tried to design a solution. The mental health problems arose because of several socioeconomic issues. Even in the case of thalassemic kids, the husbands used to blame their wives and abandon the child and mother to face social isolation and financial distress. The X-linked recessive Hemophilia made matters worse for the mothers who always carried the guilt. The “conspiracy of silence” was quite evident, and the patients were victims of stigmatization at the school and workplace. We wanted to work beyond prescription generation for this malady. GANSID: How did you overcome that challenge? Dr. Prantar: We overcame the first challenge by designing a special clinic where the patients could come and consult me directly at a prefixed frequency determined by the severity of the illness, with prior appointments and the number of patients was restricted. We could at least devote 30 minutes of our time to each patient so that we could deal with their problems with empathy and adequate discussion. To take care of the mental health issues of the patients with genetic blood disorders, we formed an organization called “The Kolkata Hematology Education and Research Initiative (KHERI) and organized several activities including music and drama workshops so that they can engage themselves in different activities and think beyond their blood disorders and can be socially integrated. You can read about our activities in an article published in the Lancet Haematology: GANSID: Is there a clinician/patient success story you would like to share with our audience? Dr. Prantar: I had a Beta Thalassemia Major patient who had been transfusion-dependent since early childhood and had also lost her mother when she was in her teens. Previously, the patients had to pay out of their pockets for their transfusion as well as iron chelation. I was able to convince the administrators that the treatment should be free of cost and that was implemented helping many such patients. The lady was very much motivated and used to help other patients and would work like a patient ambassador. She had completed her masters in English literature, and I encouraged her to get a master's degree in social welfare. When establishing thalassemia control units in different medical colleges and district hospitals of the state of West Bengal, we recruited her as a counselor as she fulfilled the educational criteria, and she has been the most empathetic counselor we ever had. Despite the regular transfusions and comorbidities, she would not only counsel all the families who were traumatized by the diagnosis of thalassemia and the natural history of the disease but help them in several ways going beyond her duties. She still works as a counselor and has been a beacon in the field of generating thalassemia awareness as well as educating the families of the patients so that they become more educated and take better care of the children with thalassemia. GANSID: How can GANSID support your work? Dr. Prantar: GANSID can support my work in various ways.

It can partner with patient advocacy groups in India so that they learn about communication, project management, and advocacy skills to highlight their conditions and raise awareness so that genetic disorders can be prevented in the country.
It can also help the twinning of centers in different parts of the world so that they may exchange their experiences and learn about their successes and failures and the ways to overcome their failures.
It can also influence multinational pharmaceutical companies to conduct more clinical trials in the field of thalassemia, hemophilia, and other genetic disorders in developing countries so that the caregivers learn how to use the new molecules and the patients also benefit and get early access to the new technology.
GANSID can develop training modules for nurses, counselors, and data managers for training online and certification.
GANSID can partner with us in developing a user-friendly app for patients with genetic disorders where the patients can upload their prescriptions and reports and record their complaints so that the complete patient journey is documented.

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Dr. Titi Adeyemo’s story

Shining Light on Clinicians Serving the Hereditary Blood Disorders Community Introducing Dr. Titi Adeyemo, a hematologist with a specialty in Sickle Cell Disease, Thalassemia, and Hemophilia Clinicians around the world play a vital role in supporting individuals living with hereditary blood disorders (HBDs). Their dedication, expertise, and compassion significantly impact the lives of patients and their families. In this blog post, we are honored to share Dr. Titi Adeyemo’s story. She is a Professor and Consultant Hematologist who has been serving the hereditary blood disorder community for 18 years. GANSID: What hereditary blood disorder/s do you treat and what leads you to that specialty area? Dr. Adeyemo: My area of specialty is sickle cell disease and other inherited blood disorders such as hemophilia. My passion for working with patients with hereditary blood disorders stems from the desire to lessen the burden of living with a hereditary blood disorder for those affected with inherited blood disorders. GANSID: Can you share a major challenge you have encountered in your practice? Dr. Adeyemo: Practicing in a resource-constrained country, many major challenges and issues are often encountered. Top on the list is the inability of many to afford the cost of basic care for their disease such as the inability to pay for blood transfusion, hydroxyurea, or routine blood tests (for those with sickle cell disease). Additionally, there is poor patient knowledge about disease management. GANSID: How did you overcome that challenge? Dr. Adeyemo: By being positive and staying motivated. You just feel good when you see the impact you make in someone’s life sometimes. Collaborating with various patient groups to arrange lectures and seminars on the basic care of inherited blood disorders. By brainstorming solutions with every patient who has a challenging care plan. My goal is to assist patients in seeking help from charitable organizations or affluent individuals, as well as identifying and enrolling them in feasible health insurance schemes when or where possible. GANSID: Is there a clinician/patient success story you would like to share with our audience? Dr. Adeyemo: I will share a patient’s management success story. Amara, 31 is no stranger to debilitating hip problems and recurrent pain crises. She had never been in a Sickle Cell Disease (SCD) care program and only sought pain relief for her crises at clinics. But when both of her hip joints started giving her a lot of pain and she had to be on many pain medications several times daily, she eventually showed up at our center for help. “My hips are giving me sleepless nights; my life has been full of pains, and it appears there is no hope she said, I am always on pain medication and was recently told that the pain meds will give me kidney failure. Is there anything else we can do?” Turns out, she never knew that as a sickle cell disease patient, she needed to be in a comprehensive care program for regular evaluation, and monitoring for chronic organ dysfunction rather than episodic visits for acute events. She had never attended a sickle cell disease clinic nor seen a specialist and only sought pain relief for pain episodes when they occurred. With counseling and education about SCD care, blood transfusion, and hydroxyurea therapy, she eventually had both hips replaced with excellent results, and no leg brace was needed afterward. She has been pain-free for the past 18 months. Putting the swing and spring back in Amara’s step with comprehensive SCD care and hip replacement surgery made a lot of difference. She has gone further and enrolled in a master’s program. For me, she is a role model of a positive attitude in rehabilitation and recovery. But too many do not have access to basic or comprehensive care, and we must do more to change that narrative. GANSID: How can GANSID support your work? Dr. Adeyemo: GANSID can help by improving awareness and the level of public knowledge about hereditary blood disorders. This will help with advocacy efforts. Concluding statement from GANSID Dr. Adeyemo's dedication has made a real difference in the lives of many patients. She is helping countless individuals manage their hereditary blood disorders, improve their quality of life, and achieve their goals. Dr. Adeyemo's story is a powerful testament to the impact that clinicians can have on the lives of patients with hereditary blood disorders. Their dedication, compassion, and expertise are essential in providing patients with the care they need and deserve. We are grateful for Dr. Adeyemo's commitment to the #HBD community and all the #clinicians who work tirelessly to support individuals and families affected by #hereditaryblooddisorders.

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Dr. Adekunle Adekile- Eastern Mediterranean Region

Dr. Adekunle Adekile, Professor, Department of Pediatrics, Faculty of Medicine, Kuwait University. Dr. Adekile graduated from the University of Ibadan Medical School, Nigeria, and had his Pediatric Residency Training at the Obafemi Awolowo University Teaching Hospital, Ile-Ife Nigeria. He was in Howard University Hospital, Washington, DC, USA, from 1980-1981 for his post-doctoral Fellowship in Pediatric Hematology, during which time he worked at the Comprehensive Sickle Cell Center under Dr. Roland Scott. He established a sickle cell clinic in the Obafemi Awolowo University Teaching Hospital on his return to Nigeria in 1981. In 1990 he was the recipient of the Fogarty International Fellowship which took him to the Medical College of Georgia, Augusta, GA, USA where he worked in Dr. Titus Huisman’s laboratory and the Comprehensive Sickle Cell Center. Dr. Adekile moved to Kuwait in 1993; he is the Chief of Pediatric Hematology at Mubarak Hospital where he looks after patients with SCD, thalassemia, and other benign hematology disorders. He is the director of the Kuwait National SCD Registry, which he established in 2011. He is active in the hematology professional circles in the Arabian Gulf. He holds a Ph.D. in Cell Biology from the University of Maastricht, Netherlands based on his thesis “Comparative Studies of Nigerian and Kuwaiti SCD patients”. His research expertise is in the phenotypic variability of the disease, especially the presentation among patients with the Arab/India haplotype and elevated Hb F. He was the founding chairman of the Nigerian SCD Network and the Sickle Cell Support Society of Nigeria. He has more than 160 publications in peer-reviewed journals. He has served as a consultant to WHO/AFRO on the management of SCD in Africa. He is the current Editor-in-Chief of HEMOGLOBIN, the International Journal of Hemoglobin Research.

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Black History Month: Honoring Black Heroes and History

As we observe Black History Month in the USA, Canada, UK and the Ireland, I take pride in acknowledging the remarkable contributions made by Black pioneers in the fight against inherited blood disorders. This month provides us with the opportunity to reflect on the significant impact Black individuals and communities have had in shaping our world, especially in advancing inherited blood disorders including sickle cell disease.

The Global Action Network for Sickle Cell & Other Inherited Blood Disorders (GANSID) is dedicated to combating along with stakeholders working in hereditary blood disorders, the many challenges faced by the affected millions of people; and we are motivated by the progress we have already made.
Nonetheless, we recognize that we are just starting the work. Inherited blood disorders have a disproportionate impact on communities of color, with Black individuals being particularly affected! As such, it is crucial that we continue to strive for a future where inherited blood disorders no longer hinder the health and well-being of individuals and families globally.

We understand the importance of diversity and inclusion in the fight against inherited blood disorders. Black scientists, doctors, and researchers have made significant contributions to this field, and we are committed to creating a space where everyone is valued and empowered to making a difference. We believe that diverse perspectives lead to better outcomes, and we are dedicated to ensuring that all voices are heard and respected.

We also recognize the importance of education and awareness in the fight against inherited blood disorders. Many people are not aware of the impact these conditions have on individuals and communities. We will continue to raise awareness and educate the public through collaborations with our member organizations, industry partners, clinicians and people with lived experiences. We will work tirelessly towards a future where inherited blood disorders are better understood and more effectively treated.

As we celebrate Black History Month, let us honor the contributions of those who have come before us and continue to work towards a brighter future. Let us celebrate the recent breakthrough therapies for sickle cell and thalassemia disorders, and stand together in the fight against inherited blood disorders and empower all for progress.

Yours in advocacy,
Mrs. Lanre Tunji-Ajayi, M.S.M

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CASGEVY: A Cure for Sickle Cell & Thalassemia Disorders

CASGEVY: A Cure for Sickle Cell & Thalassemia Disorders.

Thornton, Canada, November 17, 2023, Casgevy by the Boston-based pharmaceutical company, Vertex is the first medicine to be licensed that uses the innovative genome-editing tool CRISPR, for which its inventors (Emmanuelle Charpentier of the Max Planck Unit for the Science of Pathogens and Jennifer Doudna of the University of California, Berkeley) were awarded the Nobel Prize in Chemistry in 2020

The inventors first showed that CRISPR—which stands for clustered regularly interspaced short palindromic repeats—could edit DNA in an in vitro system in a paper published in the 28 June 2012 issue of Science. Their discovery was rapidly expanded on by many others and soon made CRISPR a common tool in labs around the world.

Today, Vertex Inc. has revolutionized treatments of hereditary blood disorders by using the CRISPR technology to bring about a cure for sickle-cell disease and transfusion-dependent β-thalassemia; and the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has authorized the use of this treatment for patients aged 12 and over after a rigorous assessment of its safety, quality, and effectiveness.

Sickle Cell Thalassemia Disorders are two hereditary blood disorders that are debilitating, painful, and life-altering. They have resulted in lifelong complications and premature death for many of the affected. Globally, many of the affected people are not identified and those identified experience a life clouded with stigma, discrimination, and poor access to quality care and treatments.

According to the CEO of the Global Action Network for Sickle Cell & Other Inherited Blood Disorders, Lanre Tunji-Ajayi, M.S.M, CASGEVY is a welcome innovation for the hereditary blood disorders community especially those living with sickle cell and the thalassemia disorders. She advised that CASGEVY is a viable cure and that everything should be done to ensure that cost will not prohibit its access to people who need it, especially in low and medium-resource countries.

Lanre Tunji-Ajayi, M.S.M also reminisced in an interview with Canadian Broadcasting Corporation (CBC) that CASGEVY is one of the dreams of her brother, Engr. Sunday Afolabi (whom she lost to preventable complications of sickle cell disease in October 1998) spoke about when he said during his lifetime “ I looked forward to a day when no one will suffer from sickle cell disease”. On this note, she urges Vertex Inc. and other pharmaceuticals working on similar technologies to ensure that the price of this breakthrough therapy is affordable in order for more people to take advantage of it.

Vertex Inc, CEO Reshma Kewalramani advised the Global Action Network for Sickle Cell and Other Hereditary Blood Disorders that “As the first CRISPR/Cas9 gene-edited treatment to be approved, this is a momentous scientific achievement, not only for Vertex but for the patients whose lives we have the potential to transform”

Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning hemoglobin. To do this, stem cells are taken out of bone marrow, edited in a laboratory, and then infused back into the patient after which the results have the potential to be life-long.

CBC INTERVIEW

REFERENCE

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Roundtable 2023 at ASH

More Pictures from ASH

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Pre-launch Consultation with Inherited Blood Disorders Organizations in Nigeria

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Prof. JM. Dangou – WHO

Prof. JM. Dangou World Health Organization (WHO) Prof. JM. Dangou has an international reputation for his work in Noncommunicable diseases (NCDs) and, in Health Diplomacy, leadership and cooperation, and management. He served at the WHO Regional Office for Africa, and currently, he is the Acting WHO Representative to the Republic of Zimbabwe. In several countries, he coordinated the WHO teams for planning, implementation, monitoring, and evaluation of policies, strategies, and health policies and plans. He was also in charge of diplomatic representation, strategic direction, and the establishment of partnerships. Before joining WHO, Professor Dangou held various positions in academia and the Ministry of Health of Senegal. He published more than 140 scientific articles, more than 80% of them in peer-reviewed journals. Jean-Marie is a Medical Doctor, specializing in Histopathology, Cytology, and Cytogenetics and, Epidemiology. He undertook professional training and continuing education in Pedagogy including e-learning, Research methodology, Communications, Global Health Diplomacy, Leadership, and Management among others.

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Dr. Adekunle Adekile – Kuwait

Adekunle Adekile, MD, PhD Kuwait University Dr. Adekile graduated from the University of Ibadan, Nigeria, and had his post-doctoral Fellowship in Pediatric Hematology at Howard University Hospital, Washington DC, USA. He was also a Fogarty International Fellow in Molecular Genetics at the Medical College of Georgia, Augusta, GA, USA. He is currently a Professor at Kuwait University, Kuwait. He was the Founding Chairman of the Nigerian Sickle Cell Disease Network and the Sickle Cell Support Society of Nigeria. Dr. Adekile’s expertise is on the clinical, hematological, and molecular aspects of SCD patients with the high HbF phenotype. As the Chief of Pediatric Hematology at Mubarak Al-Kabeer Hospital, Kuwait, Dr. Adekile looks after patients with other benign hematological disorders apart from hemoglobinopathies (SCD and thalassemia). He serves as a consultant to WHO/AFRO on producing a guidance framework for the management of SCD in Africa. He is the current Editor-in-Chief of HEMOGLOBIN, the International Journal of Hemoglobin Research.

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Dr. Laure Joseph – France

Laure JOSEPH MD

Hôpital Necker Enfants-Malades Paris

Dr. Laure Joseph has been a haematologist in the AP-HP Necker Hospital in Paris since 2015 (in the team of Prof Marina Cavazzana). She specializes in benign haematology like sickle cell disease. She is in charge of pregnancy Sickle Cell Disease women and her hospital is a reference centre for this specificity. In addition, she is involved in bone marrow transplantation and gene therapy clinical trials.

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Author - InHeBloodDisor