根据 WHO 分类，对 170 例晚期肥大细胞增多症患者的预后评分进行了比较，并进行了 C 扫描，并接受了米斯托林治疗。
Comparison of prognostic scores according to WHO classification in 170 patients with advanced mastocytosis and C-finding treated with midostaurin.
发表日期：2024 Nov
作者：
Maël Heiblig, Clément Gourguechon, Philippe Guilpain, Cristina Bulai-Livideanu, Stéphane Barete, Yannick Chantran, Julie Agopian, Fabienne Brenet, Patrice Dubreuil, Jérémie Lespinasse, Richard Lemal, Olivier Tournilhac, Louis Terriou, David Launay, Laurence Bouillet, Catharina Chatain, Ghandi Damaj, Thomas Ballul, Celine Greco, Laura Polivka, Laurent Frenzel, Cécile Meni, Hassiba Bouktit, Dina Benabou, Caroline Gaudy-Marqueste, Marie Gousseff, Edwige Le Mouel, Antoine Neel, Dana Ranta, Roland Jaussaud, Thierry Jo Molina, Julie Bruneau, Patrick Villarese, Ludovic Lhermitte, Leila Maouche-Chrétien, Marie Temple, Olivier Kosmider, Rose-Marie Javier, Fabien Pelletier, Florence Castelain, Frederique Retornaz, Quentin Cabrera, Patricia Zunic, Marie Pierre Gourin, Ewa Wierzbicka-Hainaut, Jean François Viallard, Christian Lavigne, Cyrille Hoarau, Isabelle Durieu, Sophie Dimicoli-Salazar, Jose Miguel Torregrosa-Diaz, Mathieu Wemeau, Angèle Soria, Michel Arock, Christine Bodemer, Olivier Lortholary, Olivier Hermine, Julien Rossignol
来源：
AMERICAN JOURNAL OF HEMATOLOGY
摘要：
晚期系统性肥大细胞增多症 (AdvSM) 包括异质性肥大细胞增多症亚型，并与不良预后相关。尽管米斯托林是第一个被批准用于 AdvSM 患者的酪氨酸激酶抑制剂，但长期反应有限。突变调整风险评分 (MARS)、国际肥大细胞增多症预后评分系统 (IPSM) 和系统性肥大细胞增多症全球预后评分 (GPSM) 已建立，以描述总体 AdvSM 患者的结局。然而，鉴于结果对 AdvSM 亚型的依赖性，每个亚型内的预后特征至关重要。我们的目的是根据 AdvSM 亚型使用 Harrell 预后评分一致性指数来研究预测能力。我们利用法国肥大细胞增多症参考中心的登记系统进行了一项全国范围的回顾性研究，纳入了所有接受米斯托林治疗且有 C 发现的患者。总共确定了 170 名患者：46 名侵袭性 SM (ASM)、11 名肥大细胞白血病 (MCL) 和 113 名 SM 伴相关血液肿瘤 (SM-AHN)。当与 AdvSM 亚型结合时，所有风险评分都提高了其对总生存期 (OS) 的判别价值。最佳预测值是调整后的 MARS (C-index = 0.689)，其次是 GPSM (C-index = 0.677) 和 IPSM (C-index = 0.618)。在多变量分析中，MARS 分层和 AdvSM 亚型均可预测 OS。因此，确定了 AdvSM 患者和不同中位 OS 的五个亚组：MCL 为 9.9 个月，中/高风险 SM-AHN 为 24 个月，中/高风险 ASM 为 33 个月，低风险 SM 为 58 个月-AHN，且未达到低风险 ASM (p< 0.001)。 AdvSM 亚型和 MARS 对 OS 最具预测性，应促进具体管理。© 2024 作者。 《美国血液学杂志》由 Wiley periodicals LLC 出版。
Advanced systemic mastocytosis (AdvSM) encompasses heterogeneous mastocytosis subtypes and is associated with poor outcomes. Although midostaurin was the first tyrosine kinase inhibitor to be approved for AdvSM patients, long-lasting responses are limited. The mutation-Adjusted Risk Score (MARS), the International Prognostic Scoring System for mastocytosis (IPSM) and the Global Prognostic Score for Systemic Mastocytosis (GPSM) have been established to characterize the outcomes of patients with overall AdvSM. However, given the outcome's dependency on the AdvSM subtype, prognostic characterization within each subtype is critical. We aimed to study the predictive ability using Harrell's concordance index of prognostic scores according to the AdvSM subtype. We conducted a nationwide retrospective study using the French mastocytosis reference center's registry and included all midostaurin-treated patients with C finding. Overall, 170 patients were identified: 46 aggressive SM (ASM), 11 mast cell leukemia (MCL), and 113 SM with associated hematological neoplasm (SM-AHN). All risk scores improved their discriminative value for overall survival (OS) when combined with the AdvSM subtype. The best predictive value was for adjusted MARS (C-index = 0.689), followed by GPSM (C-index = 0.677) and IPSM (C-index = 0.618). In a multivariable analysis, MARS stratification and the AdvSM subtype were both prognostic for OS. Accordingly, five subgroups of patients with AdvSM and a different median OS were identified: 9.9 months for MCL, 24 months for intermediate/high-risk SM-AHN, 33 months for intermediate/high-risk ASM, 58 months for low-risk SM-AHN and was not reached for low-risk ASM (p < 0.001). The AdvSM subtype and the MARS are the most predictive of OS and should prompt specific management.© 2024 The Author(s). American Journal of Hematology published by Wiley Periodicals LLC.