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Posttransplant Cyclophosphamide for HLA-haploidentical Transplantation in Patients With Mucopolysaccharidosis.

08:00 EDT 4th April 2018 | BioPortfolio

Summary of "Posttransplant Cyclophosphamide for HLA-haploidentical Transplantation in Patients With Mucopolysaccharidosis."

We successfully used a haploidentical transplantation protocol with posttransplant cyclophosphamide (CY) (50 mg/kg/d on days +3 and +4) for in vivo T-cell depletion in patients with mucopolysaccharidosis using reduced-intensive conditioning regimens, followed by a busulfan-based conditioning regimen, which included busulfan (12 to 16 mg/kg) and fludarabine(150 to 200 mg/m)+rabbit antihuman thymocyte globulin (7.5 to 10 mg/kg) as a conditioning regimen. Cyclosporine or tacrolimus, methotrexate, mycophenolate mofetil, and methylprednisolone were administered to prevent graft-versus-host disease (GVHD). After follow-up for a median period of 1.5 years, all 8 patients without preexisting severe comorbidities and early transplant referrals are alive, with 100% donor chimerism and excellent performance status. Only 1 patient developed chronic GVHD(II). We conclude that posttransplant CY is effective in vivo for T-cell depletion to promote full donor engraftment in patients with mucopolysaccharidosis. In addition, with posttransplant CY, the procedure reduced the rate of GVHD and the cost of transplant and improved the patients' quality of life.

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This article was published in the following journal.

Name: Journal of pediatric hematology/oncology
ISSN: 1536-3678
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Medical and Biotech [MESH] Definitions

Transplantation between individuals who share a partial haplotype match.

Mucopolysaccharidosis characterized by excessive dermatan and heparan sulfates in the urine and Hurler-like features. It is caused by a deficiency of beta-glucuronidase.

Mucopolysaccharidosis with excessive chondroitin sulfate B in urine, characterized by dwarfism and deafness. It is caused by a deficiency of N-acetylgalactosamine-4-sulfatase (arylsulfatase B).

Mucopolysaccharidosis characterized by heparitin sulfate in the urine, progressive mental retardation, mild dwarfism, and other skeletal disorders. There are four clinically indistinguishable but biochemically distinct forms, each due to a deficiency of a different enzyme.

Precursor of an alkylating nitrogen mustard antineoplastic and immunosuppressive agent that must be activated in the LIVER to form the active aldophosphamide. It has been used in the treatment of LYMPHOMA and LEUKEMIA. Its side effect, ALOPECIA, has been used for defleecing sheep. Cyclophosphamide may also cause sterility, birth defects, mutations, and cancer.

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