Study reveals breakthrough in treating children with sickle cell disease via stem cell transplant

Gurugram, Nov 13(PTI) A decade-long study by a Gurugram hospital has found success in curing Sickle Cell Disease among children through bone marrow (stem cell) transplantation, placing India among the leading nations in advanced paediatric transplant outcomes, officials said on Thursday.

The study by the Fortis Memorial Research Institute (FMRI), which was recently published in the international journal ‘Haemoglobin’, analysed 100 paediatric cases treated between 2015 and 2024.

The results demonstrated an overall survival rate of nearly 87 per cent with 96 per cent success among matched sibling donor transplants and 78 per cent 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 (SCD) in developing nations, a hospital statement said.

SCD 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 permanent cure, the statement said.

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 SCD.

“Our research shows that children in developing countries, such as 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.” According to the study, early diagnosis and timely transplant are critical for long-term survival, the statement said.

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 minimise side-effects and reduce the risk of graft-versus-host disease (GVHD) – a common post-transplant complication, the statement said.

Dr Vikas Dua, principal consultant and head, paediatric haematology, oncology and bone marrow transplant, Fortis Gurugram, said that many of these children had been living with pain, repeated hospitalisations, and dependence on transfusions.

“Today, they are leading a healthy and active life. This milestone reinforces our belief that every child deserves a chance at a normal life and that early intervention is the key to success,” Dua said.

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 safe and more widely applicable, the statement said.

Dr Rahul Bhargava, principal director, Institute of Blood Disorders and Bone Marrow Transplant, Fortis Gurugram, emphasised the broader impact of the study.

“India and Africa together carry nearly half the world's burden of SCD. 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,” Bhargava said.

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 SCD can become a reality for children worldwide,” she said.

Yashpal Rawat, VP and facility director, Fortis Gurugram, said, “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.” PTI PLB ARI