Transplant to treat rare genetic disorder

Unrelated blood cord transplant was used at Ganga Ram Hospital to treat 16-month-old Manya of Hurler’s Syndrome, a rare genetic disorder.

Unrelated blood cord transplant was used at Ganga Ram Hospital to treat 16-month-old Manya of Hurler’s Syndrome, a rare genetic disorder.

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The disease causes deposition of abnormal proteins in various body organs, leading to bone deformity, big and abnormal face, thick skin, blindness and developmental delays.

“Hurler’s syndrome is caused by a defective enzyme. The child had been undergoing enzyme therapy,” said Dr Ratna Dua Puri, consultant, department of genetic medicine at Sir Ganga Ram Hospital.

“Her parents also could not be donors as they both had the defective gene,” said Dr SP Yadav, consultant. “We then considered unrelated cord blood stem cells,” said Dr Anupam Sachdeva, Director, paediatric haematology oncology and bone marrow transplantation.

Manya underwent the double cord blood transplant on October 10. She was kept in bone marrow transplant room in isolation for a month to prevent infection but was discharged 35 days after the transplant.