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

Hematopoietic cell transplantation (HCT) has been used for more 30 years for the treatment of selected malignant and nonmalignant diseases. Traditionally, HCT for hematological disorders has relied on myeloablative conditioning before HLA-identical sibling bone marrow transplantation to correct the underlying hematological defect. Most children with hematological diseases who are referred to HCT have features that portend significant morbidity and early mortality. Among SAA patients who have HLA-identical sibling donors, younger patients with profound pancytopenia might be considered early for HCT. For others who lack sibling donors, patients who receive HCT from alternate sources have generally failed one or more courses of intensive immunosuppressive therapy and remain transfusion-dependent, some with hemosiderosis, red cell alloimmunization, and platelet transfusion refractoriness [44,46,48]. Currently, HCT for SCD is generally restricted to those who have experienced a significant sickle-related complication such as stroke, recurrent acute chest syndrome, or recurrent painful episodes [7,13]. In contrast, most reserve HCT in thalassemia for younger, Lucarelli class I, good-risk patients who have HLA-identical sibling donors, and veer away from older, high-risk thalassemics for whom transplantation is a riskier clinical intervention. For groups such as young adults with thalassemia major, HCT might become more widely applicable if its toxicity was reduced. Several approaches undergoing development include reduced-intensity conditioning and attempts to prevent GVHD. New methods to reduce the intensity and toxicity of conditioning as well as to use highly purified stem cells with the reduction in graft versus host disease may allow for the use of matched unrelated donors or haploidentical donors. This would serve to provide potentially more children who could benefit from stem cell transplantation with donors. These advances will hopefully lead to benefits for the majority of children who lack HLA-identical donors.
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PMID:New approaches to hematopoietic cell transplantation for hematological diseases in children. 1243 Jun 22

We report on a phase I/II, single arm clinical trial studying the safety and efficacy of Traditional Chinese Medicine (TCM) in patients with various chronic cytopaenic marrow diseases including myelodysplastic syndrome (MDS), myelofibrosis (MF), aplastic anaemia (AA) and thalassemia intermedia, who either have failed, are unfit for or refused currently available Western medical treatment. Patients took oral herbal concoctions according to their TCM syndromes for 24 weeks while continuing with western medical management. The median age of this group of 31 patients was 61 (26--84) years old and median disease duration was 5 years (0.3--40 years). TCM herbs were well tolerated in these patients with multiple comorbidities and previous disease-related complications. Twenty-three patients completed the study with 5 (2 with MDS, 2 with MF and 1 with SAA) achieving some degree of haematological improvement. EORTC quality of life indicators improved in more than half of patients. This small study offers positive results and provides the basis for future larger studies which should randomize patients with MDS, MF and AA managed with standard Western medical treatment to without and with upfront combinations with TCM herbs. This will conclusively define the role of TCM in the supportive management of these diseases. This study was registered with Clinicaltrial.gov as NCT01224496.
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PMID:Traditional Chinese herbal medicine in the supportive management of patients with chronic cytopaenic marrow diseases -- a phase I/II clinical study. 2174 81