Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0019045 (hemoglobinopathies)
 2,704 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

An immunologic and virologic work-up was undertaken in 425 symptom-free multitransfused patients with hemophilias or hemoglobinopathies living in France. Patients were entered into five groups according to the type of blood product they received: local factor VIII, a mixture of local and imported factor VIII, imported factor IX, local factor IX, washed red blood cells. The overall prevalence of IgG antibodies to the lymphadenopathy-associated virus (LAV) was 45%. The highest rate was observed in hemophiliacs who received factor VIII concentrates prepared from plasma collected mainly on the American continent; intermediary values were found for hemophilic patients treated with local factor VIII or factor IX concentrates; and the lowest values were found for those who were treated with washed red blood cells. Lymphadenopathy, decreased skin hypersensitivity reactions, relative lymphopenia, and altered ratio of T lymphocyte subsets occurred at significantly higher rates in patients positive for LAV antibody, although such abnormalities were also encountered in LAV serologically negative patients. A correlation between treatment intensity and immunologic disturbances was found in patients infused with factor VIII preparations, irrespective of their positive or negative LAV antibody status. This study has shown the prominent role of LAV in the occurrence of immunologic disturbances in multitransfused patients. However, allogenic or altered proteins present in factor VIII but not in factor IX concentrates seem to play a role of immunocompromising agents. The interplay between LAV and additional factors possibly leading to acquired immunodeficiency syndrome remains to be analyzed.
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PMID:Immunologic and virologic status of multitransfused patients: role of type and origin of blood products. By the AIDS-Hemophilia French Study Group. 299 80

Patients with sickle cell disease (N = 3) and thalassemia (N = 1) with high-risk features received hematopoietic stem cell transplantations (HCT) to induce stable (full or partial) donor engraftment. Patients were 9-30 years of age. Fludarabine, rabbit anti-thymocyte globulin (ATG), and 200 cGy total body irradiation were administered pre-transplant. Patients received bone marrow (N = 3) or peripheral blood stem cells (N = 1) from HLA-identical siblings, followed by mycophenolate mofetil and cyclosporine for post-grafting immunosuppression. Significant lymphopenia, but only moderate neutropenia and thrombocytopenia developed post transplant. No grade IV nonhematological toxicities or acute graft-versus-host disease (GVHD) were observed. At 3 months after transplantation, three of four patients had evidence of donor myeloid chimerism (range, 15-100%). However, after post transplant immunosuppression was discontinued, graft rejection occurred in all but one patient. This patient is now doing well 27 months post transplant with full donor engraftment. One patient died after a second transplant, and another patient experienced a stroke as her graft was being rejected. These results suggest that stable donor engraftment after nonmyeloablative HCT is difficult to achieve among immunocompetent patients with hemoglobinopathies and that new approaches will need to be developed before wider application of this transplantation method for hemoglobinopathies.
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PMID:Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after low-dose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin. 1553 1