World’s First Successful Half-Matched Transplant for Rare Genetic Disorder

Chennai: MGM Cancer Institute has successfully performed a complex TCR alpha beta depleted haploidentical bone marrow transplant (BMT) on a 12-year-old girl with Bloom Syndrome, a rare genetic disorder. The transplant, using stem cells from her younger brother, marks the first documented success of such a procedure in a pediatric patient worldwide.

Complex Procedure

The patient, diagnosed with Bloom Syndrome at the age of 10, had developed Monosomy 7 and Myelodysplastic Syndrome (MDS), a disorder that can progress to leukemia. Finding a suitable donor was challenging, and the medical team had to adapt the standard stem cell collection protocol and conditioning regimen to ensure the best possible outcome for both siblings.

Successful Outcome

The transplant team, led by Dr. M. Deenadayalan, achieved a successful outcome, with the patient now crossing one-year post-transplant with full immunological recovery and 100% donor chimerism. The patient is attending school regularly and participating in daily activities like other children her age.

Groundbreaking Achievement

This achievement is a testament to the expertise of MGM Cancer Institute’s BMT team and has been published in Pediatric Blood and Cancer, a leading peer-reviewed journal in pediatric hematology and oncology. The case highlights the institute’s commitment to providing cutting-edge treatment options for complex and rare conditions.