Gene/Protein Disease Symptom Drug Enzyme Compound
Pivot Concepts:   Target Concepts:
Query: UMLS:C0039730 (thalassemia)
 10,305 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Bone marrow transplant (BMT) recipients are routinely reimmmunized with the childhood vaccine series after transplantation excluding the live attenuated vaccines. In this study, the clinical and serologic responses to measles, mumps and rubella (MMR) vaccine in children after BMT was assessed. Twenty-two BMT recipients were vaccinated with MMR II (MSD). All were at least 2 years post-BMT and without GVHD. Their underlying conditions were leukemia (11), aplastic or Fanconi's anemia (7), thalassemia (3) and metabolic disease (1). All were allogeneic transplants with matched related donors. The mean age at transplantation was 6.9 years. There were no reported adverse effects of the vaccination. Antibody status for MMR was determined using commercial assays (IFA and ELISA) on paired specimens. The mean interval between transplantation and vaccination was 48 months. Pre-vaccination, no BMT recipient was sero-positive for all three, but 23% were positive for measles, 31% for mumps and 14% for rubella. Post-vaccination, 68% of BMT recipients were sero-positive for all three, with 77% for measles, 87% for mumps and 91% for rubella. Therefore, MMR vaccination at 2 years or later after BMT in paediatric recipients without GVHD was safe and significantly increased the proportion sero-positive for MMR.
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PMID:Response to measles, mumps and rubella vaccine in paediatric bone marrow transplant recipients. 872 67

Bone marrow transplantation (BMT) performance can be limited by a lack of ideal donors, and the role of alternative donor hematopoietic cell transplantation in thalassemia is not well established. Here we used a new treatment protocol (Pc 26.1) in 16 thalassemia patients to perform BMT using phenotypically HLA-identical or 1-antigen-mismatched relatives (related donors [RDs]). We compared these results with HLA-matched sibling (matched sibling donors [MSDs]) BMT in 66 patients. The entire RD group and 88% of MSD group had sustained engraftment. Rejection incidence was 0% in the RD and 12% (95% confidence interval [95% CI], 6%-21%) in MSD groups (P = .15), with respective thalassemia-free survival probabilities of 94% (95% CI, 63%-99%) and 82% (95% CI, 70%-89%) (P = .24). Transplant-related mortality was 6% (95% CI, 1%-26%) in the RD group and 8% (95% CI, 3%-16%) in the MSD group (P = .83). The intensified new protocol was not associated with increased nonhematologic toxicity. The present data show that the Pc 26.1 preparative regimen allows thalassemia patients to safely undergo BMT from RDs who are not HLA-matched siblings, with transplant outcomes similar to patients with MSD grafts.
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PMID:Bone marrow transplantation for thalassemia from alternative related donors: improved outcomes with a new approach. 2396 44