A second chance at life through haploidentical stem cell transplantation


Attendees of the Paediatric Haploidentical Bone Marrow Transplantation Programme's 10th anniversary celebration include (front row, from right) One Hope Charity and Welfare chairman Datuk Chua Sui Hau, Prof Hany, UM Pro-Chancellor Toh Puan Dr Hajah Aishah Ong and UMMC director Prof Dr Mohd Zulkiflee Abu Bakar, as well as the transplant team members, support staff, parents and children who had undergone the procedure. — UMMC

It has been a decade since the Paediatric Haploidentical Bone Marrow Transplantation Programme at Universiti Malaya Medical Centre (UMMC) began.

When it was first established, few could have imagined the impact it would eventually have.

Bone marrow transplantation – more accurately known as haematopoietic stem cell transplantation (HSCT) – is among the most complex treatments in modern medicine.

It offers a potential cure for many life-threatening blood disorders, including leukaemia, lymphoma, aplastic anaemia, inherited immune deficiencies, thalassaemia and several rare genetic diseases.

Historically, stem cell donors were limited to siblings or unrelated donors who had full human leukocyte antigen (HLA) compatibility with the patient.

Over time, new techniques to perform HSCT with “half-matched donors”, also known as haplo-identical donors, were developed.

This greatly increased the number of patients who could benefit from the procedure as parents or half-matched siblings were now suitable alternative donors.

Lifesaving innovation

Behind every successful transplant lies a deeply personal story.

In 2016, UMMC senior consultant paediatric oncologist Prof Dr Hany Ariffin was referred a one-year-old baby with severe combined immunodeficiency (SCID), also known as Bubble Boy Disease.

Ravaged by recurrent infections, he weighed only 5kg and was oxygen dependent.

He desperately needed a new immune system via stem cell transplantation.

But being an only child and with no matched donor, the outlook seemed bleak.

With backing from the hospital, Prof Hany adapted a protocol from Johns Hopkins Hospital in the United States, allowing the boy’s “half-matched” father to be the bone marrow donor.

After a stormy 149 days, the child recovered.

He is now 11 years old and thriving.

This success was then followed by more referrals from within and outside Malaysia.

Among the programme’s “graduates” are children who had never attended school because of severe congenital immune disorders, adolescents battling aggressive leukaemia, and those born with inherited blood diseases requiring lifelong transfusions.

Many families arrive with tragic stories of having lost older children before a diagnosis was made and the curative option of transplant was offered to the surviving child.

Over 10 years, the UMMC team steadily expanded its expertise while continuously refining clinical protocols, improving infection prevention strategies and adopting internationally accepted standards of care.

The management of each case is individually tailored to the patient, with details such as underlying illnesses and nutritional status addressed beforehand so that the child enters the transplantation process in an optimal state.

The result is a programme capable of delivering highly specialised care that was once unavailable, and is still currently very much inaccessible to many children in need.

While not all children who have undergone the procedure survive, nevertheless, every milestone – eating independently, walking without assistance, returning to school or work, receiving routine childhood vaccinations again – becomes a celebration.

Advancing medical research

Beyond patient care, the programme has also contributed to clinical research and professional training.

Studies examining transplant outcomes, infection prevention, long-term survivorship and quality of life have helped improve care for future patients.

Healthcare professionals have shared their findings nationally and internationally, strengthening collaborations with transplant centres across the region.

Prof Hany, who is also UMMC’s Paediatric Haematology-Oncology and Blood & Marrow Trans-plantation Division head, is currently chairperson of the Viva-Asia Bone Marrow Transplant Consortium.

This consortium organises two “schools” annually for training and international collaboration in the Asia-Pacific region.

These efforts ensure that patients benefit not only from today’s medical knowledge, but also from continuous improvements driven by research and innovation.

Medicine continues to evolve rapidly.

New approaches – including cellular therapies, gene editing, precision medicine and improved supportive care – promise to make transplantation even safer and more effective.

For UMMC’s HSCT team, the next decade represents an opportunity not only to perform more transplants, but also to improve every aspect of the patient journey – from earlier diagnosis and donor availability to survivorship care and long-term quality of life.

The vision remains clear: ensuring that more patients, regardless of where they live or their financial circumstances, have access to lifesaving treatment.

A legacy of life

Celebrating 10 years is not simply about commemorating a number.

It is about honouring the half-matched miracles that turned parents into donors, children into saviours, and rejection into remission.

The UMMC Bone Marrow Transplantation Unit and its research laboratory have been fortunate enough to receive support from local charities, as well as government research grants.

The costs associated with HSCT are often way beyond the means of many patients.

For these patients and their families, the help from Pusat Pungutan Zakat − Majlis Agama Islam Wilayah Persekutuan, One Hope Charity and Welfare, various corporations and many individual donors is gratefully acknowledged.

As the Paediatric Haploidentical Bone Marrow Transplantation Programme celebrates a decade of service and more than 100 successful transplants, its greatest achievement cannot be measured solely by statistics.

Its true legacy lives in school classrooms filled once again with children who almost never returned, in parents who no longer fear when their child will face the “time’s up” moment, and in families who have been given the precious gift of ordinary days.

Looking to the future, the UMMC Paediatric HSCT team hopes the programme will continue to make cures available and accessible for all children who require it.

Parents who were previously told there was “no chance” for their children to undergo transplantation are now met with a half-match lifeline.

This represents a second shot at life, not just for the patient, but also for the entire family.

After all, the greatest success in medicine is by sometimes simply helping a child live long enough to dream about tomorrow.

Dr Syaza Zafirah Ab Rahman is a consultant paediatric haematologist at UMMC. For more information, email starhealth@thestar.com.my. The information provided is for educational and communication purposes only, and should not be considered as medical advice. The Star does not give any warranty on accuracy, completeness, functionality, usefulness or other assurances as to the content appearing in this article. The Star disclaims all responsibility for any losses, damage to property or personal injury suffered directly or indirectly from reliance on such information.

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