The Paediatric Haploidentical Bone Marrow Transplantation Programme at Universiti Malaya Medical Centre (UMMC) has marked its 10th anniversary since its inception in 2016. The programme, which specializes in haematopoietic stem cell transplantation (HSCT), provides potentially curative treatment for serious blood disorders such as leukaemia, lymphoma, aplastic anaemia, inherited immune deficiencies, and thalassaemia.
Traditionally, HSCT required donors to be fully human leukocyte antigen (HLA) compatible, usually from siblings or unrelated matched donors. However, advances in transplantation techniques have allowed the use of “half-matched” or haploidentical donors, expanding the pool of eligible donors to include parents and partially matched siblings. This development has significantly increased access to life-saving transplants for patients lacking fully matched donors.
One of the earliest and notable cases handled by the programme involved a one-year-old child with severe combined immunodeficiency (SCID), or Bubble Boy Disease, who was dependent on oxygen and weighed just 5 kilograms. Lacking a fully matched donor, the child’s only option was a transplant from his haploidentical father, following a protocol adapted from Johns Hopkins Hospital in the United States. After a critical 149-day recovery period, the boy has since thrived and is now 11 years old. This outcome helped establish the viability of the programme, leading to increased referrals across Malaysia and internationally.
Over the past decade, UMMC’s multidisciplinary team has refined transplantation protocols, enhanced infection prevention measures, and incorporated internationally recognized standards of care. Individualized patient management includes pre-transplant assessments to optimize nutritional status and address coexisting conditions. While not all transplants achieve successful long-term outcomes, milestones such as independent feeding, mobility, school attendance, and resumption of routine vaccinations are celebrated as significant progress in patient recovery.
Beyond clinical care, the programme has contributed to research on transplant outcomes, infection prevention, long-term survivorship, and quality of life. The team’s work has been shared through national and international collaborations, strengthening regional expertise. Prof Dr Hany Ariffin, UMMC’s senior consultant paediatric oncologist and head of the Paediatric Haematology-Oncology and Blood & Marrow Transplantation Division, also chairs the Viva-Asia Bone Marrow Transplant Consortium, which facilitates training and collaboration across the Asia-Pacific region.
Sustaining the programme has required substantial support from local charities, government research grants, and individual donors, as the costs of HSCT often exceed the financial capabilities of many families. Organisations such as Pusat Pungutan Zakat-Majlis Agama Islam Wilayah Persekutuan and One Hope Charity and Welfare have been instrumental in providing aid.
As the programme moves forward, the UMMC team aims to broaden access to transplantation, improve donor availability, and enhance comprehensive survivorship care. Emerging medical advances including cellular therapies, gene editing, and precision medicine are expected to improve the safety and effectiveness of treatment.
Celebrating over 100 successful paediatric haploidentical transplants, the programme’s legacy is reflected not only in clinical achievements but also in the restored hopes and improved lives of children and families. For many patients previously deemed ineligible for transplant, haploidentical transplantation offers a renewed opportunity for survival and a chance to envision a healthy future.