At SGPGI Lucknow, father donates stem cells to save son with rare genetic disorder

Dr Sayan Sinha Roy, assistant professor in the department of hematology, said hematopoietic stem cell transplantation (HSCT), commonly known as a bone marrow transplant, is “currently the only established treatment capable of stopping the progression of X-ALD,” provided it is performed before permanent neurological damage occurs.

The Sanjay Gandhi Postgraduate Institute of Medical Sciences (SGPGIMS) has successfully performed a haplo-identical bone marrow transplant on a five-year-old boy suffering from X-linked Adrenoleukodystrophy (X-ALD), a rare inherited genetic disorder that mainly affects young boys and damages the brain, spinal cord and adrenal glands. The life-saving procedure was made possible after the child’s father donated stem cells, SGPGIMS said on Sunday.

SGPGI Lucknow (Sourceds)

Conducted under the National Programme for Rare Diseases (NPRD), the transplant is SGPGIMS’s first successful bone marrow transplant for X-ALD and, according to the institute, the first such successful procedure in North India.

A haplo-identical transplant is a bone marrow or stem cell transplant using a half-matched family donor, usually a parent, when a fully matched donor is unavailable.

Dr Sayan Sinha Roy, assistant professor in the department of hematology, said hematopoietic stem cell transplantation (HSCT), commonly known as a bone marrow transplant, is “currently the only established treatment capable of stopping the progression of X-ALD,” provided it is performed before permanent neurological damage occurs.

The transplant was led by Dr Rajesh Kashyap, head of the department of hematology, with support from experts in hematology, medical genetics and laboratory medicine.