Browsing by Subject "Thalassemia"
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Item Open Access Eighth Annual International Umbilical Cord Blood Transplantation Symposium, San Francisco, California, June 3-5, 2010.(Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2011-02) Laughlin, Mary; Kurtzberg, Joanne; McMannis, John; Petz, LawrenceItem Open Access Prevalence of left ventricular diastolic dysfunction by cardiac magnetic resonance imaging in thalassemia major patients with normal left ventricular systolic function.(BMC cardiovascular disorders, 2019-11-06) Chinprateep, Benjaporn; Ratanasit, Nithima; Kaolawanich, Yodying; Karaketklang, Khemajira; Saiviroonporn, Pairash; Viprakasit, Vip; Krittayaphong, RungrojBACKGROUND:The leading cause of mortality of thalassemia major patients is iron overload cardiomyopathy. Early diagnosis with searching for left ventricular diastolic dysfunction before the systolic dysfunction ensued might yield better prognosis. This study aimed to define the prevalence of the left ventricular diastolic dysfunction (LVDD) in thalassemia major patients with normal left ventricular systolic function and the associated factors. METHODS:Adult thalassemia major patients with normal left ventricular systolic function who were referred for cardiac T2* at Siriraj Hospital - Thailand's largest national tertiary referral center - during the October 2014 to January 2017 study period. Left ventricular diastolic function was defined by mitral valve filling parameters and left atrial volume index using CMR. Patients with moderate to severe valvular heart disease, pericardial disease, or incomplete data were excluded. Baseline characteristics, comorbid diseases, current medication, and laboratory results were recorded and analyzed. RESULTS:One hundred and sixteen patients were included, with a mean age of 27.5 ± 13.5 years, 57.8% were female, and 87.9% were transfusion dependent. Proportions of homozygous beta-thalassemia and beta-thalassemia hemoglobin E were 12.1 and 87.9%, respectively. The baseline hematocrit was 26.3 ± 3.3%. The prevalence of LVDD was 20.7% (95% CI: 13.7-29.2%). Cardiac T2* was abnormal in 7.8% (95% CI: 3.6-14.2%). Multivariate analysis revealed age, body surface area, homozygous beta-thalassemia, splenectomy, heart rate, and diastolic blood pressure to be significantly associated with LVDD. CONCLUSIONS:LVDD already exists from the early stages of the disease before the abnormal heart T2 * is detected. Homozygous beta-thalassemia and splenectomy were strong predictors of LVDD. These data may increase awareness of the disease, especially in the high risk groups.Item Open Access Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.(Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2011-09) Ruggeri, Annalisa; Eapen, Mary; Scaravadou, Andromachi; Cairo, Mitchell S; Bhatia, Monica; Kurtzberg, Joanne; Wingard, John R; Fasth, Anders; Lo Nigro, Luca; Ayas, Mouhab; Purtill, Duncan; Boudjedir, Karim; Chaves, Wagnara; Walters, Mark C; Wagner, John; Gluckman, Eliane; Rocha, Vanderson; Eurocord Registry; Center for International Blood and Marrow Transplant Research; New York Blood CenterWe examined the efficacy of unrelated cord blood (CB) transplantation in children with thalassemia (n = 35) and sickle cell disease (n = 16), using data reported to 3 registries. Donor-recipient pairs were matched at HLA-A and -B (antigen level) and DRB1 (allele level) in 7 or HLA mismatched at 1 (n = 18), 2 (n = 25), or 3 loci (n = 1). Transplant conditioning was myeloablative (n = 39) or reduced intensity (n = 12). Neutrophil recovery with donor chimerism was documented in 24 patients; 11 patients developed grade II-IV acute graft-versus-host disease (aGVHD) and 10 patients, chronic GVHD (cGVHD). Overall survival (OS) and disease-free survival (DFS) were 62% and 21% for thalassemia and 94% and 50% for sickle cell disease (SCD), respectively. In multivariate analysis, engraftment rate (hazard ratio [HR] 2.2, P = .05) and DFS (HR 0.4, P = .01) were higher with cell dose >5 × 10(7)/kg. The 2-year probability of DFS was 45% in patients who received grafts with cell dose >5 × 10(7)/kg and 13% with lower cell dose. Primary graft failure was the predominant cause of treatment failure occurring in 20 patients with thalassemia and 7 patients with SCD. Primary graft failure was fatal in 5 patients with thalassemia. These results suggest that only CB units containing an expected infused cell dose >5 × 10(7)/kg should be considered for transplantation for hemoglobinopathy.