Report of 100 hurler syndrome patients receiving 115 bone marrow transplants

C. Peters, W. Krivit, J. E. Wagner

Research output: Contribution to journalArticle

Abstract

Hurler syndrome (MPS 1 H), deficiency of a-L-iduronidase enzyme activity, leads to severe dementia, characteristic facial features, hepatosptonomegaly, cardiac disease, severe skeletal abnormalities, hydrocephalus, and premature death usually by 5 years of age due to accumulation of glycosaminoglycan substrate. Engraftment following bone marrow transplantation (BMJ) provides enzyme and leads to major improvement in symptoms and longevity (COGENT III. 1995; 2540). Various myeloablatrve, marrow manipulation, graft versus host disease (GVHD) prophylaxis regimens and stem cell sources were used. Results according to bone marrow donor type are shown below. Donor pts/BMTs aBVH(%) cGVHD(%) Engraft(%) Survival(%) Identical° 27/30 32 0 95 74 Related° 26/27 46 20 64 54 Unrelated+ 40/51 30 18 63 49 Unmlated 7/7 43 0 100 63 acute GVHD: Grade II-IV, chronic GVHD: extensive; °in preparation; + in press, BLOOD 1996; † BLOOD 1994; 84:394a (abstract) Variation in treatment protocols precludes identification of the optimal preparative methodology. These results are best described as Phase l/ll studies. To Improve the outcomes for future patients, permanent engraftment rates must be enhanced and GVHD must be minimized or eliminated. An international cooperative study is needed to address these issues.

Original languageEnglish (US)
Number of pages1
JournalExperimental Hematology
Volume24
Issue number9
StatePublished - Dec 1 1996

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