Sirolimus and mycophenolate mofetil as GVHD prophylaxis in myeloablative, matched-related donor hematopoietic cell transplantation

Autor: Robert S. Negrin, Robert Lowsky, David B. Miklos, Laura Johnston, Sally Arai, Mareike Florek, Judith A. Shizuru, Janice M. Brown, Philip W. Lavori, K Sheehan, J S McCune, Randall Armstrong, Ginna G. Laport
Rok vydání: 2011
Předmět:
Male
Transplantation Conditioning
medicine.medical_treatment
Graft vs Host Disease
Disease
Hematopoietic stem cell transplantation
Mycophenolate
Gastroenterology
0302 clinical medicine
immune system diseases
GVHD prophylaxis
Child
Hematopoietic Stem Cell Transplantation
Hematology
Middle Aged
3. Good health
Portal vein thrombosis
surgical procedures
operative
Child
Preschool
Hematologic Neoplasms
030220 oncology & carcinogenesis
Acute Disease
Female
Unrelated Donors
Immunosuppressive Agents
medicine.drug
Adult
medicine.medical_specialty
Adolescent
Article
Mycophenolic acid
03 medical and health sciences
Internal medicine
medicine
Humans
Transplantation
Homologous
T regulatory cell
Sirolimus
Transplantation
business.industry
mycophenolate mofetil
Mycophenolic Acid
medicine.disease
Surgery
business
Follow-Up Studies
030215 immunology
Zdroj: Bone marrow transplantation
ISSN: 1476-5365
0268-3369
DOI: 10.1038/bmt.2011.104
Popis: We investigated sirolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis in patients with advanced hematological malignancies receiving myeloablative hematopoietic cell transplantation (HCT) from HLA-identical sibling donors. On the basis of pre-study stopping rules, the trial was closed to accrual after enrollment of 11 adult patients. In all, 7 of the 11 patients received BU-containing preparative regimens. Sirolimus was discontinued in three patients because of the toxicity-related events of severe sinusoidal obstructive syndrome, portal vein thrombosis, altered mental status and in one patient because of the risk of poor wound healing. In all, 6 of the 11 patients developed grade II-IV acute GVHD (AGVHD) a median of 15.5 days post HCT. Two of three patients with grade IV AGVHD had sirolimus discontinued by 9 days post HCT. All patients responded to AGVHD therapy without GVHD-related deaths. There were two non-relapse- and two relapse-related deaths. At a median follow-up of 38 months (2-47 months), 7 of 11 patients were alive without disease. MMF and sirolimus GVHD prophylaxis did not reduce the risk of AGVHD, however, there were no GVHD-related deaths. The severe toxicities in the patients receiving the BU-containing preparative regimens limited the continued use of sirolimus and MMF for the prevention of AGVHD.
Databáze: OpenAIRE
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