Clinical predictors for the phenotypic heterogeneity of severe hemophilia A in China / 南方医科大学学报

<p><b>OBJECTIVE</b>To observe the phenotypic heterogeneity of severe hemophilia A in China and investigate the clinical factors for defining the severity of the clinical presentations.</p><p><b>METHODS</b>The data including the age of first bleeding and first joint bleeding, bleeding frequency, the number of joint deformities and body mass index (BMI) were collected from 223 patients with severe hemophilia A (FVIII:C≤2%).</p><p><b>RESULTS</b>The median age at first bleeding was 1 year (range: 0-35 years). The percentages of patients with first bleeding age<1 year, ≥2 years and ≥6 years were 44.3% (94/212), 34.4% (73/212), and 10.8% (23/212), respectively. The median age at first joint bleeding was 2.25 years. The percentages of patients with first joint bleeding age ≤1 year, ≥2 years and ≥6 years were 25.5% (24/94), 57.4% (54/94), and 18.1% (17/94), respectively. The percentage of patients who did not have joint bleeding was 7.4% (7/94). The median annual bleeding frequency was 24 per year (range: 1-120), and the proportion of patients with annual bleeding episodes of less than 6 times was 12.9%. Only 24.7% of the patients were free of any joint deformities. Analysis showed that milder cases had older ages of first bleeding and first joint bleeding than the severe cases, and the milder cases had also significantly lower BMI.</p><p><b>CONCLUSION</b>The age of first bleeding and first joint bleeding and BMI may predict the clinical severity of hemophilia A in China in the early stage.</p>

Analysis of infection-related mortality after allogeneic hematopoietic stem cell transplantation in patients with refractory/relapse acute leukemia / 南方医科大学学报

<p><b>OBJECTIVE</b>To investigate infection-related mortality (IRM) after allogeneic hematopoietic stem cell transplantation in patients with refractory/relapse acute leukemia.</p><p><b>METHODS</b>We conducted a retrospective analysis of 127 patients with refractory/relapse acute leukemia and investigated the incidence, causes and risk factors of IRM.</p><p><b>RESULTS</b>Sixty-seven of the patients died after the transplantation. The 5-year overall survival and disease-free survival was (35.2∓5.3)% and (30.8∓5.6)% among these patients, respectively. IRM occurred in 28.3% (36/127) of the patients. Multivariate analysis showed that grade II-IV acute graft-versus-host diseases (aGVDH, P=0.049, OR=3.017) and post-transplant invasive fungal infection (P=0.032, OR=3.223) were independent risk factors of IRM.</p><p><b>CONCLUSION</b>As a common cause of transplant-related mortality, IRM is more frequent in cases of refractory/relapse acute leukemia than in cases with a standard risk profile, and effective prophylaxis and treatment of severe GVHD remain currently the primary measures for reducing post-transplant IRM.</p>