Cytoreductive therapy in 108 adults with systemic mastocytosis: Outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine
Cytoreductive therapy in 108 adults with systemic mastocytosis: Outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine
复制标题
DOI：
10.1002/ajh.21561
复制
发表时间：
2009-12-01
影响因子：
12.8
通讯作者：
Tefferi, Ayalew
中科院分区：
文献类型：
--
作者：
Lim, Ken H.;Pardanani, Animesh;Tefferi, Ayalew
全身性肥大细胞增多症（SM）的细胞减少治疗包括几种药物，其个体优点尚未得到很好的表征。我们回顾性研究了108名梅奥诊所的患者，他们符合2008年who的SM诊断标准，并接受了至少一种细胞减少药物治疗。可评估干扰素- α联合或不联合强的松（ifn - α）、羟脲（HU）、甲磺酸伊马替尼（IM）或2-氯脱氧腺苷（2-CdA）治疗反应的患者人数分别为40人、26人、22人和22人。根据最近公布的一致标准，相应的总体（主要）应答率分别为53%（18%）、19%（0%）、18%（9%）和55%（37%）。在惰性SM、侵袭性SM和与另一克隆血流变非肥大细胞谱系疾病（SM- ahnmd）相关的SM中，ifn - α的总有效率分别为60%、60%、45%，HU为0,0,21%，IM为14%、50%、9%，2-CdA为56%、50%、55%。ifn - α治疗的患者中肥大细胞介质释放症状的缺失和2- cda治疗的患者中循环未成熟骨髓细胞的存在预示着较差的反应。TET2突变状态不影响治疗反应。尽管这四种细胞减减剂的主要缓解率仍不理想，HU主要用于SM- ahnmd患者，但目前的研究倾向于将2-CdA或IFN-a作为SM的一线治疗方法，并确定可能对此类治疗有反应的患者。点。中华血液学杂志，2009,34(4):790-794。(C) 2009 Wiley-Liss, Inc。
Cytoreductive therapy in systemic mastocytosis (SM) includes several drugs whose individual merit has not been well characterized. We retrospectively studied 108 Mayo Clinic patients who met the 2008 WHO diagnostic criteria for SM and received at least one cytoreductive drug. The numbers of patients who were evaluable for response to treatment with interferon-alpha with or without prednisone (IFN-alpha), hydroxyurea (HU), imatinib mesylate (IM) or 2-chlorodeoxyadenosine (2-CdA) were 40, 26, 22, and 22, respectively. The corresponding overall (major) response rates, according to recently published consensus criteria, were 53% (18%), 19% (0%), 18% (9%), and 55% (37%). The respective overall response rates in indolent SM, aggressive SM and SM associated with another clonal hematological nonmast cell lineage disease (SM-AHNMD) were 60%, 60%, 45% for IFN-alpha, 0, 0, 21% for HU, 14%, 50%, 9% for IM and 56%, 50%, 55% for 2-CdA. The absence of mast cell mediator release symptoms in IFN-alpha-treated patients and presence of circulating immature myeloid cells in 2-CdA-treated patients predicted inferior response. TET2 mutational status did not influence treatment response. Although the major response rates with these four cytoreductive agents were still suboptimal and HU was mainly used in patients with SM-AHNMD, the current study favors 2-CdA or IFN-a as first-line current therapy in SM and identifies patients who are likely to respond to such therapy. Am. J. Hematol. 84:790-794, 2009. (C) 2009 Wiley-Liss, Inc.