Gurugram, 8th November 2025: In a landmark advancement for Indian healthcare, doctors at FMRI, Gurugram, have reported exceptional success in curing children suffering from Sickle Cell Disease (SCD) through Bone Marrow (stem cell) transplantation, a development that places India among the leading nations in advanced paediatric transplant outcomes.
The decade-long study, published in the international journal Haemoglobin, analysed 100 paediatric cases treated between 2015 and 2024. The results demonstrate an overall survival rate of nearly 87%, with 96% success among matched sibling donor transplants and 78% success among half-matched (haploidentical) family donor transplants. These outcomes are among the best reported globally, marking a significant advance in the management of Sickle Cell Disease in developing nations.
Sickle Cell Disease is an inherited blood disorder that affects millions of children worldwide, particularly in India and sub-Saharan Africa, where nearly half of the global cases occur. The condition leads to severe anemia, recurrent pain crises, stroke, organ damage and a shorter life expectancy. Until now, treatment options were largely limited to symptom control through medications and blood transfusions. Stem cell transplantation (also known as bone marrow transplant) replaces the defective bone marrow with healthy stem cells from a compatible donor, offering a permanent cure.
Dr. Swati Bhayana, Lead Author of the study and Consultant, Paediatric Haematology, Oncology and Bone Marrow Transplant at Fortis Gurugram, said: “This is a ray of hope for families living with Sickle Cell Disease. Our research shows that children in developing countries like India and across Africa can achieve survival rates comparable to the best centres in the world when given timely access to advanced care. These results prove that cure is possible, even in resource-limited settings.”
The study revealed that early diagnosis and timely transplant are critical for long-term survival. Performing the procedure before the onset of severe complications like stroke or organ damage dramatically improves outcomes. The Fortis team achieved these results using advanced transplant protocols that minimize side effects and reduce the risk of graft-versus-host disease (GVHD) – a common post-transplant complication.
Dr. Vikas Dua, Principal Consultant & Head, Paediatric Haematology, Oncology and Bone Marrow Transplant, Fortis Gurugram, added “Many of these children had been living with pain, repeated hospitalizations, and dependence on transfusions. Today, they are leading healthy, active lives. This milestone reinforces our belief that every child deserves a chance at a normal life and that early intervention is the key to success.”
The study also noted significant progress in haploidentical (half-matched) transplants, which use parental donors when a full sibling match is unavailable. The use of reduced-toxicity conditioning and post-transplant cyclophosphamide (PTCy) protocols lowered complication rates, making the treatment safer and more widely applicable.
Dr. Rahul Bhargava, Principal Director, Institute of Blood Disorders and Bone Marrow Transplant, Fortis Gurugram, emphasized the broader impact: “India and Africa together carry nearly half the world’s burden of Sickle Cell Disease. By developing cost-effective, safe, and scalable transplant protocols, we are showing that cutting-edge medical innovation doesn’t have to be limited to the developed world. Our goal is to ensure that every child, regardless of geography or income, has access to a cure.”
Dr Sohini Chakraborty, Senior Consultant – Pediatric Hematology, Oncology and Bone Marrow Transplant, Fortis Gurugram said “The findings underscore the importance of awareness, collaboration, and early diagnosis. With improved donor registries, better infection control, and stronger post-transplant care, doctors at Fortis are confident that the cure for Sickle Cell Disease can become a reality for children worldwide.”
VP & Facility Director, Fortis Gurugram, said; “At Fortis, we believe that innovation must serve humanity. This breakthrough reflects our mission to combine world-class technology with compassion, making lifesaving treatments available and affordable for families in India, Africa and beyond. The success of this decade-long effort reflects not just medical excellence, but also Fortis Healthcare’s commitment to expanding access to advanced care across regions.”
This achievement marks a significant milestone for Indian healthcare, as FMRI becomes one of the few centres globally to demonstrate long-term, successful outcomes for Sickle Cell Disease transplants in children. With continued research, collaboration, and awareness, such cures could soon reach every child who needs them, regardless of geography or income.
Summary of the Study
Title of the Study: Encouraging Outcomes of Hematopoeitic Stem Cell Transplantation (HSCT) in Pediatric Sickle Cell Disease : A Decade-Long Experience from the Developing World
Background and Significance: Sickle cell disease is the most prevalent hemoglobinopathy, affecting around 300,000 newborns globally each year, with significant morbidity and reduced life expectancy.
- HSCT is currently the only curative treatment for SCD, but challenges include donor availability, socio-economic factors, transplant failures, and complications like graft-versus-host disease (GVHD).
- There is no standardized conditioning regimen for SCD patients undergoing HSCT, and data on outcomes from non-Western countries are limited.
Study Design and Patient Demographics
- This retrospective cohort study included 100 pediatric patients diagnosed with SCD who underwent HSCT between January 2015 and December 2024.
- Of these, 92 patients were evaluated, with 55 receiving HLA-identical sibling transplants and 37 haploidentical transplants.
- The median follow-up time was 31.6 months, with a median age of 7.5 years among participants.
Transplant Outcomes
- Overall survival (OS) was 86.9% in the cohort, with significantly higher rates in matched sibling donor (MSD) transplants (96.4%) compared to haploidentical transplants (78.3%).
- Event-free survival (EFS) was reported at 77%, with 91.2% of patients achieving stable engraftment.
- The study noted improvements in haploidentical transplant outcomes over time, particularly after adopting reduced toxicity conditioning regimens.
Graft-Versus-Host Disease and Complications
- The cumulative incidence of acute GVHD was 26%, with 8.4% experiencing chronic GVHD at two years post-transplant.
- Complications included viral reactivations in 18 patients, with systemic cytomegalovirus (CMV) reactivations being the most common.
- Other toxicities included mucositis and endothelial complications, with some patients requiring total parenteral nutrition.
Discussion and Implications
The study highlights the effectiveness of HSCT in pediatric SCD patients, particularly with MSD transplants, and emphasizes the need for improved access to HSCT in resource-limited settings. It suggests that early referral for transplantation and optimized post-transplant care can enhance survival rates.
- The findings contribute valuable data to the global understanding of HSCT outcomes for SCD, particularly in low- and middle-income countries.
