MGM Cancer Institute has set a global milestone by saving the life of a 12-year-old girl from Chennai through a complex bone marrow transplant (BMT). The procedure, known as TCR alpha beta depleted haploidentical bone marrow transplant, was performed using life-saving stem cells from her younger brother. This marks the world’s first documented success of such a transplant in a pediatric patient with Bloom Syndrome.
A Rare and High-Risk Diagnosis
As per the press release, the girl was diagnosed with Bloom Syndrome at the age of 10, a rare genetic disorder that significantly increases the risk of cancer and bone marrow failure. Her condition worsened with the development of Monosomy 7, a chromosomal abnormality involving the loss of one copy of chromosome 7, and Myelodysplastic Syndrome (MDS), where bone marrow fails to produce healthy blood cells.
Since MDS can rapidly progress to leukemia, doctors emphasized that a bone marrow transplant was both critical and urgent to save her life.
Overcoming Donor Challenges
Finding a donor proved extremely difficult. Neither fully matched family donors nor unrelated donors were available, and both parents carried the genetic mutation. After detailed genetic evaluation, the medical team identified her younger brother as the safest donor since he did not carry the mutation.
However, his young age and lower body weight created a new challenge. The doctors had to adapt the standard stem cell collection protocol to safely harvest enough stem cells without endangering him.
Tailored Treatment for a Fragile Patient
The transplant team also faced the challenge of modifying the conditioning regimen for the patient. Children with Bloom Syndrome are highly sensitive to chemotherapy and cannot withstand standard high-dose treatments. Therefore, the team designed a customized protocol to ensure effectiveness while minimizing toxicity.
These careful adjustments required meticulous planning, precision, and a multidisciplinary approach to protect both siblings and achieve success.
Leadership and Expertise
The BMT procedure was led by Dr. M. Deenadayalan, HOD and Clinical Lead, Department of Paediatric Haematology, Oncology, Blood and Marrow Transplantation.
He explained, “Bloom Syndrome is a rare genetic disorder, and in this case, it was further complicated by Monosomy 7 and Myelodysplastic Syndrome—a combination so uncommon that only a handful of cases have been reported worldwide. We are proud that we could save this young girl, who was facing one of the most complex and rare medical conditions. She has now completed one year post-transplant with full immunological recovery and 100% donor chimerism, which confirms the transplant’s success. To our knowledge, this is the world’s only documented successful TCR alpha beta depleted haploidentical BMT in a child with Bloom Syndrome.”
A Healthy New Beginning
Today, the young girl is back in school and actively participating in daily life like other children her age. Although she requires regular follow-up to monitor her long-term health, her recovery has been remarkable.
Her case has gained global recognition and was published in Pediatric Blood and Cancer, one of the world’s leading peer-reviewed journals in pediatric hematology and oncology, underscoring its international clinical significance.