Hurler syndrome surgery

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]

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Overview

Bone marrow transplantation (BMT) and umbilical cord blood transplantation (UCBT) can successfully treat the mucopolysaccharidoses. Abnormal physical characteristics, except for those affecting the skeleton and eyes, can be improved, and neurologic degeneration can often be halted. BMT and UCBT are high-risk procedures with high rates of morbidity and mortality. However, they are the only treatments that have the potential to cure the disease.

Surgery

Bone Marrow Transplantation

• These procedures lead to replacement of mutated/ non-functional cells of the bone marrow with functional cells.
• Functional new cells act as a source for the deficient enzyme.
• Transplant has the best outcome when its done as soon as the diagnosis is established.
• Children who receive a transplant early enough can have normal or near-normal mental development.
• If the disease has progressed causing organ damage, the child is at high risk of developing life-threatening complications from transplant.
• Transplants cannot undo the damage done to nervous system.
• Risks of transplant
  • Graft versus host disease
  • Graft failure
  • Pulmonary hemorrhage

Umbilical Cord Blood Transplantation

• This plays a crucial role when suitable donor is not available for transplant.
• In a study between 1995 and 2002, quite convincing results were obtained with cord blood transplants.
• Cord blood transplantation favorably altered the course of disease.