Investigation on long-term complications of glucocorticoid in treatment of Diamond-Blackfan anemia

Abstract

Objective To collect and summarize long-term complications of glucocorticoid (GC) in treatment of Diamond-Blackfan anemia (DBA). Methods A total of 17 DBA patients, treated with GC more than 1 year from December 2009 to November 2012 in Being Children's Hospital, Capital Medical University, were retrospectively investigated.The data of general information, drug treatment, treatment response, height and body mass in different the-rapy periods, and therapy related adverse reaction were collected.The data entry and the statistical analysis were performed using SPSS 16.0 software. Results Seventeen cases which fulfilled the research criteria were enrolled.The 58.8 percent of cases (10/17) began GC therapy from the age younger than 6 months.The 76.4 percent of the cases(14/17) started prednisone therapy with the dosage≥2 mg/(kg·d), and the median time of maintenance therapy with this dosage was 2 months (1-5 months). The median time of prednisone dosage greater than 0.5 mg/(kg·d) was 6 months (3-48 months). Patients were divided into 2 groups at the beginning of therapy according to whether their age was younger or older than 6 months.The median height of younger age group was -1.0 SD (-3.5-1.0 SD) of corresponded age- sex- standard height at the beginning of prednisone therapy, and was dropped to -3.5 SD(-3.5--2.0 SD)after half year treatment.For older age group, it was 0.0 SD(-1.5-2.0 SD)and -0.5 SD (-1.5-0.5 SD) respectively.During the therapy, there were 1 fracture, 2 measles pneumonia, 3 pneumonia, 3 hi-rsuitisms, 5 thrushes, and 12 central obesity cases. Conclusions GC related adverse reaction might appear when app-lying prednisone for the treatment of DBA in long term.It was suggested that GC therapy should start after 6 months old if possible, and the duration of 0.5 mg/(kg·d) GC treatment should be reduced as short as possible. Key words: Diamond-Blackfan anemia; Glucocorticoid; Complication; Growth status