Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0018133 (graft-versus-host disease)
 18,032 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Bone marrow transplantation has become the modality of choice for a number of malignant conditions. One of the primary causes of morbidity and mortality following bone marrow transplantation is graft-versus-host disease (GVHD). Moderate to severe acute GVHD affects 9% to 35% of patients undergoing standard allogeneic bone marrow transplantation. The incidence of chronic GVHD is approximately 40% to 50%. In our experience at Johns Hopkins Oncology Center, patients with stages 2, 3, and 4 acute GVHD had median survivals of only 5.4, 3.6, and 2.5 months, respectively, despite treatment. Patients with chronic GVHD do not fare much better. Their overall 10-year mortality rate remains high at 42%. Significant failure rates and toxicities have been associated with all available therapeutic options for GVHD. Pentostatin (Nipent; SuperGen, San Ramon, CA) is currently used to treat a variety of hematologic malignancies. In addition to its antineoplastic effects, considerable immunosuppressive properties have been reported. Pentostatin affects the immune system by decreasing lymphocyte number and function. Its immunosuppressive effect has promise for the treatment of GVHD and warrants further study.
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PMID:An old drug for a new disease: pentostatin (Nipent) in acute graft-versus-host disease. 1087 57

Adult-onset leukoencephalopathy with spheroids and pigmented glia (ALSP) is an autosomal dominant leukoencephalopathy caused by mutations in colony stimulating factor 1 receptor (CSF1R). Here we report clinical and imaging outcomes following allogeneic haematopoietic stem cell transplantation (HSCT) in two patients with ALSP at the University of California, San Francisco between January 2016 and December 2017. Patient 1 proceeded to transplantation at age 53 with a haplo-identical sibling donor. Patient 2, whose sister and mother had died of the disease, proceeded to transplantation at age 49 with a 12/12 human leukocyte antigen-matched unrelated donor. Both patients received reduced intensity conditioning regimens. At 28 and 26 months post-HSCT, respectively, both patients were alive, without evidence of graft-versus-host disease, with major infection at 1 year in one and new-onset seizures in the other. In both cases, neurological worsening continued post-HSCT; however, the progression in cognitive deficits, overall functional status and gait impairment gradually stabilized. There was continued progression of parkinsonism in both patients. On brain MRI, within 1 year there was stabilization of T2/FLAIR abnormalities, and after 2 years there was complete resolution of abnormal multifocal reduced diffusion. In summary, after >2 years of follow-up, allogeneic HSCT in ALSP led to interval resolution of diffusion MRI abnormalities, stabilization of T2/FLAIR MRI abnormalities, and partial clinical stabilization, supportive of treatment response. Allogeneic HSCT may be beneficial in ALSP by providing a supply of bone marrow-derived brain-engrafting myeloid cells with donor wild-type CSF1R to repopulate the microglial niche.
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PMID:Allogeneic HSCT for adult-onset leukoencephalopathy with spheroids and pigmented glia. 3184 Jul 44