Review of disease-related complications and management in adult patients with thalassemia: A multi-center study in Thailand.

Disease-related complications and management are different among patients with thalassemia. This study was aimed to review the prevalence, clinical risk factors for the complications and the management in patients with thalassemia in Thailand. A multicenter cross-sectional study was conducted in patients with thalassemia aged ≥ 18 years old. Thalassemia-related complications and management were reviewed. The clinical parameters significantly associated with the complications were analyzed by logistic regression methods. The prevalence of thalassemia-related complications was 100% in patients with transfusion-dependent thalassemia (TDT) and 58.8% in patients with non-transfusion-dependent thalassemia (NTDT). Advanced age was statistically associated with extramedullary hematopoiesis in both TDT and NTDT patients. Splenectomy was a significant risk factor for pulmonary hypertension in both groups of patients. Severe iron overload started earlier in patients with TDT than NTDT and was associated with diabetes mellitus (adjusted odds ratio (AOR) = 6.2, p-value = 0.02). Disease-related complications are more prevalent in patients with TDT than patients with NTDT. Splenectomy and advanced age were important risk factors for developing major complications in both groups. Early screening and management for specific disease-related complications should be considered in patients with thalassemia according to their clinical risk factors.

Effect of acetylsalicylic acid on thalassemia with pulmonary arterial hypertension.

OBJECTIVE: To compare pulmonary artery systolic pressure (PASP) between thalassemic patients with pulmonary arterial hypertension (PAH) for whom acetylsalicylic acid (ASA) was and was not prescribed after 1 year. METHODS: A retrospective cohort study was conducted at the hematological outpatient clinic at Chiang Rai Hospital, Chiang Rai, Thailand. All new cases of thalassemia with PAH from January 2007 to January 2012 were studied at the first month and at 12 months. The patients were classified into two groups. In one group, ASA 81 mg daily was prescribed for 1 year, whereas in another group no ASA was prescribed, due to its contraindications, which included bleeding, gastrointestinal side effects, and thrombocytopenia. PASP, estimated by a Doppler echocardiography, was measured by the same cardiologist. Propensity score adjustment was used to control confounding variables by indication and contraindication. Multivariable regression analysis was used to evaluate the effects of ASA. RESULTS: Of the 63 thalassemia patients with PAH, there were 47 (74.6%) in the ASA group and 16 (25.4%) in the no ASA group. ASA, as compared with no ASA, did not significantly reduce PASP (adjusted difference -0.95; 95% confidence interval -16.99 to 15.10; P=0.906). CONCLUSION: Low-dose ASA may not have a beneficial effect on PASP after 1 year of treatment of PAH in thalassemia.

Benefits of chronic blood transfusion in hemoglobin E/ß thalassemia with pulmonary arterial hypertension.

OBJECTIVE: The aim of the research reported here was to compare pulmonary artery systolic pressure (PASP) and 6-minute walk distance after 1 year of follow-up in hemoglobin E/ß thalassemia (E/ß-Thal) with pulmonary arterial hypertension (PAH) patients who received chronic blood transfusions versus those who received occasional transfusions. METHODS: A nonrandomized clinical trial was conducted at the Hematological Outpatient Clinic of Chiang Rai Hospital, Thailand. All adult cases of E/ß-Thal with PAH (defined as PASP >35 mmHg by Doppler echocardiography) were evaluated and followed for the next 12 months. The patients were classified into two groups by patient preference. Group 1 patients received chronic blood transfusions - one to two units of leukocyte-poor packed red cells every 2-4 weeks - over 1 year to maintain pre-transfusion hemoglobin levels of ≥7.0 g/dL. Group 2 patients received occasional transfusions over the course of 1 year, with more than 4 weeks between transfusions. All patients were treated with iron chelation when serum ferritin levels were ≥1,000 µg/dL. PASP and the 6-minute walk distance were evaluated at baseline and at 6 and 12 months. Propensity score adjustment was used to control for confounding by indication and contraindication. Multivariable regression analysis was used to evaluate the effects of chronic blood transfusion. RESULTS: There were 16 (53.3%) patients in Group 1 and 14 (46.7%) in Group 2. At 12 months, patients in Group 1 had a greater reduction in PASP than those in Group 1 (adjusted mean difference, -16.83; 95% confidence interval, -26.35 to -7.32; P=0.001). The 6-minute walk distance at 12 months in Group 1 patients was greater than that in Group 2 patients (adjusted mean difference, 46.55; 95% confidence interval, 18.08 to 75.02; P=0.001). CONCLUSION: This study found evidence that chronic blood transfusions may have beneficial effects in PAH in thalassemia patients over 1 year.

Clinical indicators for pulmonary arterial hypertension in thalassemia.

OBJECTIVE: To explore clinical indicators for pulmonary arterial hypertension (PAH) in thalassemia (Thal). MATERIAL AND METHOD: A study was conducted in thalassemia patients at Chiang Rai Hospital, Chiang Rai, Thailand. Pulmonary artery systolic pressure (PASP) was determined by doppler echocardiography and PAH was defined as PASP > 35 mmHg. Patient characteristics were extracted from medical records. Characteristics of patients with and without PAH were compared. Risk indicators were explored with logistic regression analysis. RESULTS: Two hundred twenty four patients were included, 144 E/beta-Thal, 37 homozygous beta-Thal and 43 Hb H disease. There were 65 patients (29.0%) with PAH, 53 (81.5%) with E/beta-Thal, 8 (12.3%) with homozygous beta-Thal and 4 (6.2%) with Hb H disease. In a multivariable analysis, features significantly associated with PAH were E/beta-Thal (OR = 1.98, 95% CI; 1.29-3.01) and post splenectomy status (OR = 2.36, 95% CI; 1.17-4.73). CONCLUSION: Significant indicators for PAH in thalassemia were E/beta-Thal and post splenectomy status.