HomeScienceGenetics2-year-old Pakistani girl successfully treated for extremely rare genetic disease in UAE

2-year-old Pakistani girl successfully treated for extremely rare genetic disease in UAE

Dr. Aabideen (second from the right) and his staff cheer Eman in the hospital.

Burjeel Medical City successfully performed a bone marrow transplant (BMT) on a 2-year-old Pakistani girl with PGM3 deficiency SCID, an extremely rare genetic disorder that predisposes babies to serious infections from the moment they are born.

Dr. Zainul Aabideen, head of the pediatric hematology, oncology and bone marrow transplant department at Burjeel Medical City, and his team successfully performed the procedure on Eman, whose 4-year-old brother donated his bone marrow for his little sister.

When Eman was born two years ago, she appeared to be a healthy and happy baby. However, within a few days she developed an infection, which was treated with antibiotics. Little did her parents know that their apparently healthy baby was very sick. Over the next few months, Eman developed multiple infections of the ear, chest, skin, and mouth. These viral, bacterial and fungal infections led to multiple hospitalizations and many courses of antibiotics. By the time she was 9 months old, her parents had consulted infectious disease experts who ordered genetic testing based on her history. The tests confirmed that Eman had PGM3 deficiency SCID.

Bone marrow Pakchidl Paramedics cheer for Eman and her parents at the hospital. WAM

At Burjeel Medical City, Dr. Aabideen and his team conducted further evaluations and tests to determine if she was suitable for a bone marrow transplant, the only treatment for this condition. Since Eman and her brother were a good match, it was decided that her brother would be the donor.

“Both the patient and the donor underwent the necessary examinations. All the tests at Burjeel Medical City showed that her brother was the best donor for her and that he was suitable to donate bone marrow for his sister,” said Dr. Aabideen.

SCID or Severe Combined Immunodeficiency Disease is the most serious form of Primary Immunodeficiency Disease (PID), a rare genetic disorder that causes life-threatening problems with the immune system.

Eman was admitted on July 7 and the team successfully performed the BMT on July 18. She was kept in hospital for a month for post-transplant care, while the medical team continuously monitored her improvement. The post-BMT period is crucial, with the one-month and three-month milestones reflecting the patient’s progress. Even after 100 days, Eman had not developed any health problems or recurrent infections. Once she crossed this important milestone, the medical team at Burjeel Medical City called the transplant very successful.

WAM

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