Clinical features predict responsiveness to imatinib in platelet-derived growth factor receptor-alpha-negative hypereosinophilic syndrome.
Clinical features predict responsiveness to imatinib in platelet-derived growth factor receptor-alpha-negative hypereosinophilic syndrome.
复制标题
作者：
Khoury P;Desmond R;Pabon A;Holland-Thomas N;Ware JM;Arthur DC;Kurlander R;Fay MP;Maric I;Klion AD
除了FIP 1 L1-PDGFRA融合基因的存在外，对临床定义的嗜酸性粒细胞增多综合征（HES）中伊马替尼反应的预测因子知之甚少。患有FIP 1 L1-PDGFRA-髓样肿瘤（FP; n =12）、具有≥4项提示髓样肿瘤标准的PDGFRA阴性HES（MHES; n =10）或具有<4项髓样肿瘤标准的类固醇难治性PDGFRA阴性HES（SR; n = 5）的受试者入组伊马替尼治疗的前瞻性研究（NCT 00044304：在clinicaltrials.gov注册）。主要结局为1个月时嗜酸性粒细胞计数<1.5 × 109/L和临床症状改善。评估了伊马替尼的临床、分子和骨髓反应。根据前瞻性研究中使用的标准，对18例接受伊马替尼（300-400 mg每日一次≥ 1个月）治疗的临床定义的HES受试者的回顾性队列进行分类。总体而言，FP组（n = 16）、MHES组（n = 13）和SR组（n = 16）的伊马替尼缓解率分别为100%、54%和0%。存在≥ 4个骨髓特征是缓解的唯一预测因素。完全缓解≥ 18个月后，8例FP和1例MHES受试者逐渐减少伊马替尼剂量并停用。7例受试者（6例FP，1例MHES）在停止治疗后仍处于缓解状态，中位时间为29个月（范围14-36）。MHES的临床特征可预测PDGFRA阴性HES中伊马替尼的反应。
With the exception of the presence of the FIP1L1-PDGFRA fusion gene, little is known about predictors of imatinib response in clinically-defined hypereosinophilic syndrome (HES). Subjects with FIP1L1-PDGFRA-myeloid neoplasm (FP; n =12), PDGFRA-negative HES with ≥4 criteria suggestive of a myeloid neoplasm (MHES; n =10), or steroid-refractory PDGFRA-negative HES with <4 myeloid criteria (SR; n = 5) were enrolled in a prospective study of imatinib therapy (NCT00044304: registered at clinicaltrials.gov). The primary outcome was an eosinophil count <1.5 × 109/L at one month and improvement of clinical symptoms. Clinical, molecular, and bone marrow responses to imatinib were assessed. A retrospective cohort of 18 subjects with clinically-defined HES who received imatinib (300–400 mg daily ≥ 1 month) were classified according to the criteria used in the prospective study. Overall, imatinib response rates were 100% in the FP group (n = 16), 54% in the MHES group (n = 13) and 0% in the SR group (n = 16). The presence of ≥ 4 myeloid features was the sole predictor of response. After ≥ 18 months in complete remission, imatinib was tapered and discontinued in 8 FP and 1 MHES subjects. Seven subjects (6 FP, 1 MHES) remain in remission off therapy for a median of 29 months (range 14–36). Clinical features of MHES predict imatinib response in PDGFRA-negative HES.
登录
查看更多内容
影响因子：
6.5
作者：
Metzgeroth, Georgia;Walz, Christoph;Reiter, Andreas
通讯作者：
Reiter, Andreas
影响因子：
158.5
作者：
Apperley, JF;Gardembas, M;Goldman, JM
通讯作者：
Goldman, JM
DOI：
10.3324/haematol.2009.016345
发表时间：
2010-05-01
期刊：
HAEMATOLOGICA-THE HEMATOLOGY JOURNAL
影响因子：
--
作者：
Erben, Philipp;Gosenca, Darko;Reiter, Andreas
通讯作者：
Reiter, Andreas
影响因子：
3.4
作者：
Helbig, Grzegorz;Hus, Marek;Kyrcz-Krzemien, Slawomira
通讯作者：
Kyrcz-Krzemien, Slawomira
影响因子：
14.2
作者：
Ogbogu, Princess U.;Bochner, Bruce S.;Butterfield, Joseph H.;Gleich, Gerald J.;Huss-Marp, Johannes;Kahn, Jean Emmanuel;Leiferman, Kristin M.;Nutman, Thomas B.;Pfab, Florian;Ring, Johannes;Rothenberg, Marc E.;Roufosse, Florence;Sajous, Marie-Helene;Sheikh, Javed;Simon, Dagmar;Simon, Hans-Uwe;Stein, Miguel L.;Wardlaw, Andrew;Weller, Peter F.;Klion, Amy D.
通讯作者：
Klion, Amy D.