Analysis of the clinical features, treatment options and clinical outcomes of Fanconi anemia patients

doi:10.19538/j.ek2017050608

Abstract：Objective To analyze the clinical characteristics of children with Fanconi anemia(FA) and their responses to different drug treatments,and to study the clinical outcomes of different drug treatments.Methods A total of 43 children with definite diagnosis of FA in Institute of Hematology and Blood Diseases Hospital,Chinese Academy of Medical Sciences & Peking Union Medical College (From October 2003 to December 2014) were included.Fisher's exact test was used to analyze the potential difference of different treatments.Results Of 43 cases,the median age of onset was 5 years (50 days to 14 years),and there were 23 male cases(53.5％) and 20 female cases(46.5％).The male to female ratio.was 1.15 ∶ 1.There were 30 cases (69.8％) of malformation,and the incidence of hand deformity was the highest (34.3％).No family history was observed in 27 cases(62.8％).The most common first symptoms were anemia and thrombocytopenia.The authors further analyzed the efficacy of cases with the follow-up time≥6 months,and found that there was significant efficacy difference between the androgen +/-glucocorticoid/rabbit anti-human thymocyte immunoglobulin (ATG)+cyclosporin A group and androgen group as well as androgen+glucocorticoid groups (P ＜ 0.05).Conclusion The incidence of malformations in children with FA is inconsistent with some other international reports.The deformity or family history does not exist in all children with FA.The poor clinical prognosis and progression of FA are associated with the treatment of cyclosporin A.