MGM Cancer Institute Marks a Milestone with First Successful Pediatric Bone Transplant

The diagnosis of Myelodysplastic Syndrome (MDS) was made in a 10-year-old, revealing defective blood cell production in the bone marrow, together with the chromosomal defect known as Monosomy 7.

A 12-year-old girl with Bloom Syndrome, a hereditary disorder that increases cancer risk and causes bone marrow failure, was saved from certain death thanks to a groundbreaking and intricate bone marrow transplant performed at MGM Cancer Institute.

The transplant involved stem cells donated by the patient’s younger brother, using a specialized method known as TCR alpha-beta depleted haploidentical bone marrow transplantation.The institute reports this as the first documented instance of a successful pediatric transplant globally.

The youngster, diagnosed at age 10, was reported to have Myelodysplastic Syndrome (MDS), marked by defective blood cell production in the bone marrow, together with Monosomy 7, a chromosomal anomaly.

Without therapy, MDS had a significant chance of turning into leukemia, so the transplant was both necessary and urgent.

The hospital reported that finding a suitable donor was challenging, as no fully matched family or unrelated donors were available, and both parents were carriers of the mutation. After genetic testing, they found that her younger brother was a good donor because he didn’t have the mutation. However, because he was younger and weighed less, changes had to be made to the stem cell collection technique to make sure that the harvest was safe but enough. Also, the transplant conditioning strategy had to be changed because children with Bloom Syndrome are very sensitive to chemotherapy and can’t handle normal regimens.

The life-saving procedure was completed successfully thanks to the expertise of a dedicated medical team led by Dr. M. Deenadayalan.

It is said that the patient has gone back to school and is able to do things every day. She will continue to receive medical follow-up to keep an eye on her long-term health.

The case was featured in Pediatric Blood and Cancer, a respected journal dedicated to childhood blood disorders and cancers.

This underscores the significance of the case for doctors worldwide. Dr. Deenadayalan stated, “Bloom Syndrome alone is rare, but combined with Monosomy 7 and Myelodysplastic Syndrome, it becomes an extraordinarily unusual case, noted only in limited reports across the world.”

We are glad that we were able to save the life of a young girl who had this very unusual and complicated disease. She is now more than a year post-transplant and has fully recovered her immune system.She has achieved complete donor chimerism, as all of her blood cells are now being generated from the donor’s healthy stem cells.

This marks an important indication of the transplant’s success and is believed to be the first reported case of a successful haploidentical bone marrow transplant in a child with Bloom Syndrome.