Is Pegfilgrastim Safe and Effective in Congenital Neutropenia? An Analysis of the French Severe Chronic Neutropenia Registry
Is Pegfilgrastim Safe and Effective in Congenital Neutropenia? An Analysis of the French Severe Chronic Neutropenia Registry
复制标题
DOI：
10.1002/pbc.22147
复制
发表时间：
2009-12-01
影响因子：
3.2
通讯作者：
Donadieu, Jean
中科院分区：
文献类型：
--
作者：
Beaupain, Blandine;Leblanc, Thierry;Donadieu, Jean
目标。研究培非格司亭治疗先天性中性粒细胞减少症（CN）患者的疗效和安全性。方法.入组法国重度CN登记研究的17例患者接受了培非格司亭治疗。结果培非格司亭引入时的中位年龄为19.1岁（范围3.9-52.3岁）。在14例病例中，在中位6.9年的GCSF治疗后，培非格司亭替代了GCSF（非格司亭或来格司亭）。培非格司亭的剂量通常为每次注射一整瓶（5名儿童除外，他们接受1/6至1/2瓶），导致剂量在50至286 μ g/kg之间。培非格司亭方案范围为每7天注射2次至每30天注射1次，并有停药期。培非格司亭首次和末次给药之间的中位间隔为0.8年（0.01-4.1年）。培非格司亭组的中性粒细胞绝对计数增加趋势比GCSF组更强，但差异无统计学显著性。在培非格司亭治疗期间，两名患者发生严重感染，另外两名患者发生复发性ENT感染。9例患者报告骨痛，1例患者发生贫血和血小板减少症（WHO 111级），1例患者发生慢性荨麻疹（WHO 111级），1例GDSlb患者单次注射培非格司亭后发生呼吸窘迫，15天后死亡。在上次更新时，10例患者停止接受培非格司亭治疗，7例患者仍在接受培非格司亭治疗。结论与常规GCSF相比，培非格司亭更难用于先天性中性粒细胞减少症，不良事件更频繁，有时疗效较差。儿科血液癌症2009;53：1068-1073。(C)2009威利-利斯公司
Aims. To examine the efficacy and safety of pegfilgrastim in patients with congenital neutropenia (CN). Methods. Seventeen patients enrolled in the French Severe CN Register received pegfilgrastim. Results. Median age at pegfilgrastim introduction was 19.1 years (range 3.9-52.3 years). In 14 cases pegfilgrastim replaced GCSF (filgrastim or lenograstim), after a median of 6.9 years of GCSF therapy. The dose of pegfilgrastim was usually one full vial per injection (except in five children, who received 1/6 to 1/2 a vial), resulting in a dose of between 50 and 286 mu g/kg. The pegfilgrastim schedule ranged from two injections every 7 days to one injection every 30 days, with treatment-free periods. The median interval between the first and last dose of pegfilgrastim was 0.8 years (0.01-4.1 years). The absolute neutrophil count tended to increase more strongly on pegfilgrastim than on GCSF, but the difference was not statistically significant. During pegfilgrastim therapy, a severe infection occurred in two patients and recurrent ENT infections in two other patients. Bone pain was reported by nine patients, anemia and thrombocytopenia occurred in one patient (WHO grade 111), chronic Urticaria Occurred in one patient (WHO grade 111), and a single pegfilgrastim injection was followed by respiratory distress and death 15 days later in a patient with GDSlb. At the last update, 10 patients had stopped receiving pegfilgrastim and seven patients were still receiving pegfilgrastim. Conclusion. Compared to conventional GCSF, pegfilgrastim is more difficult to use in congenital neutropenia, with more frequent adverse events and sometimes poor efficacy. Pediatr Blood Cancer 2009;53: 1068-1073. (C) 2009 Wiley-Liss, Inc.