Life-threatening pulmonary hemorrhages post bone marrow transplantation in Hurler syndrome. Report of three cases and review of the literature.
Hurler syndrome (MPS-IH) is an autosomal recessive mucopolysaccharide storage disorder caused by deficiency of lysosomal alpha-L-iduronidase (IDU) enzyme activity. This results in accumulation of heparan sulfate and dermatan sulfate substances. Untreated children develop progressive developmental deterioration and multisystem morbidity with a median survival of 5 years. Allogeneic bone marrow transplantation (BMT) is the only long-lasting treatment that ameliorates or halts the aggressive course of the disease. Pulmonary hemorrhage (PH) is an unusual complication of BMT and has not been previously reported in MPS-IH post-BMT. We report three children with MPS-IH with life-threatening PH around the time of engraftment. All needed intensive-care support and one child developed recurrent PH that required prolonged ventilation.
Gassas
A
A
Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, 555 University Avenue, Toronto, Ontario, Canada M5G 1X8.
Sung
L
L
Doyle
J J
JJ
Clarke
J T R
JT
Saunders
E Fred
EF
D016026:Bone Marrow Transplantation / Q000009:adverse effects*; D016638:Critical Illness; D005260:Female; D006470:Hemorrhage / Q000209:etiology*; D006801:Humans; D007223:Infant; D008171:Lung Diseases / Q000209:etiology* / Q000628:therapy; D008297:Male; D008059:Mucopolysaccharidosis I / Q000628:therapy*; D012121:Respiration, Artificial; D016896:Treatment Outcome