Comparison of prognostic scores according to WHO classification in 170 patients with advanced mastocytosis and C-finding treated with midostaurin,American Journal of Hematology

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Comparison of prognostic scores according to WHO classification in 170 patients with advanced mastocytosis and C-finding treated with midostaurin
American Journal of Hematology ( IF 10.1 ) Pub Date : 2024-09-17 , DOI: 10.1002/ajh.27478
Maël Heiblig ₁ , Clément Gourguechon ₂ , Philippe Guilpain ₃ , Cristina Bulai-Livideanu ₄ , Stéphane Barete _{5,

6} , Yannick Chantran _{7,

8,

9} , Julie Agopian ₁₀ , Fabienne Brenet ₁₁ , Patrice Dubreuil ₁₁ , Jérémie Lespinasse ₁₂ , Richard Lemal ₁₃ , Olivier Tournilhac ₁₃ , Louis Terriou ₁₄ , David Launay ₁₅ , Laurence Bouillet ₁₆ , Catharina Chatain ₁₆ , Ghandi Damaj ₁₇ , Thomas Ballul _{6,

18} , Celine Greco ₁₉ , Laura Polivka _{17,

20,

21} , Laurent Frenzel _{6,

18} , Cécile Meni ₆ , Hassiba Bouktit ₆ , Dina Benabou ₆ , Caroline Gaudy-Marqueste ₂₂ , Marie Gousseff ₂₃ , Edwige Le Mouel ₂₄ , Antoine Neel ₂₅ , Dana Ranta ₂₆ , Roland Jaussaud ₂₇ , Thierry Jo Molina ₂₈ , Julie Bruneau _{18,

28} , Patrick Villarese ₂₉ , Ludovic Lhermitte _{29,

30} , Leila Maouche-Chrétien ₁₈ , Marie Temple _{31,

32} , Olivier Kosmider _{31,

32} , Rose-Marie Javier ₃₃ , Fabien Pelletier ₃₄ , Florence Castelain ₃₅ , Frederique Retornaz ₃₆ , Quentin Cabrera ₃₇ , Patricia Zunic ₃₇ , Marie Pierre Gourin ₃₈ , Ewa Wierzbicka-Hainaut ₃₉ , Jean François Viallard ₄₀ , Christian Lavigne ₄₁ , Cyrille Hoarau ₄₂ , Isabelle Durieu _{43,

44} , Sophie Dimicoli-Salazar ₄₅ , Jose Miguel Torregrosa-Diaz ₄₆ , Mathieu Wemeau ₄₇ , Angèle Soria ₄₈ , Michel Arock ₄₉ , Christine Bodemer _{20,

21} , Olivier Lortholary ₆ , Olivier Hermine _{6,

19} , Julien Rossignol _{6,

18}

Affiliation

Hematology Department, Centre Hospitalier Lyon Sud, Pierre-Bénite, France.
Department of Hematology, Amiens University Hospital, Amiens, France.
CEREMAST, the Department of Internal Medicine-Multi-organ Diseases, Saint-Eloi University Hospital, Montpellier University, Montpellier, France.
CEREMAST Toulouse, Dermatology Departmen, CHU de Toulouse, Toulouse, France.
Sorbonne Université, APHP, Unité Fonctionnelle de Dermatologie, Groupe Hospitalier Pitié-Salpêtrière-C. Foix, Paris, France.
French Reference Center for Mastocytosis (CEREMAST), Necker-Enfants Malades University Hospital, APHP, Paris, France.
Department of Biological Hematology, Pitié-Salpêtrière Hospital, DMU BioGem, AP-HP, Sorbonne University, Paris, France.
Department of Biological Immunology, Saint-Antoine Hospital, DMU BioGem, AP-HP, Sorbonne University, Paris, France.
Health Environmental Risk Assessment (HERA) Team, Centre of Research in Epidemiology and Statistics (CRESS), Inserm/INRAE, Faculty of Pharmacy, Université de Paris, Paris, France.
Association Française pour les Initiatives de Recherche sur le Mastocyte et les Mastocytoses (AFIRMM), Paris, France.
Centre de Recherche en Cancérologie de Marseille (CRCM) INSERM U1068, CNRS UMR7258, AMU U105, Département d'Onco-Hématologie-Immunologie, Institut Paoli-Calmettes, Marseille, France.
eXYSTAT, Malakoff, France.
Adult Clinical Hematology, Clermont-Ferrand University Hospital, INSERM CIC501, EA 7453-Université Clermont Auvergne, Clermont-Ferrand, France.
INSERM, CHU Lille, Department of Internal Medicine and Clinical Immunology, U1286 - INFINITE - Institute for Translational Research in Inflammation, University Lille, Lille, France.
INSERM, CHU Lille, Service de Médecine Interne et Immunologie Clinique, Centre de référence des maladies autoimmunes systémiques rares du Nord et Nord-Ouest de France (CeRAINO), U1286 - INFINITE - Institute for Translational Research in Inflammation, University Lille, Lille, France.
Internal Medicine Department, Grenoble Alpes University Hospital, Univ. Grenoble Alpes, T-RAIG unit, CNRS, UMR 5525, VetAgro Sup, Grenoble INP, CHU Grenoble Alpes, TIMC, Grenoble, France.
Haematology Institute, Normandy University School of Medicine, Caen, France.
Imagine Institute, INSERM U1163, Necker-Enfants Malades University Hospital, Paris University, Paris, France.
CEREMAST, the Department of Pain and Palliative Care Unit, Necker-Children's Hospital, AP-HP, Paris Centre University, Paris, France.
Department of Dermatology, Reference Center for Genodermatoses (MAGEC), Necker Hospital for Sick Children, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris Cité University, Paris, France.
CEREMAST, the Department of Hematology, Necker-Children's Hospital, AP-HP, Paris Centre University, Paris, France.
Dermatology and Skin Cancer, Department, Aix-Marseille University, APHM, Marseille, France.
Department of Internal Medicine, Bretagne Atlantique Hospital, Vannes, France.
Department of Internal Medicine and Clinical Immunology, Rennes University Hospital, Rennes, France.
CEREMAST, the Department of Internal Medicine, Hôtel-Dieu University Hospital, Nantes, France.
Department of Haematology, Nancy University Hospital, Nancy, France.
Department of Internal Medicine and Clinical Immunology, Vandoeuvre-lès-Nancy, France.
Department of pathology, Necker-Children's Hospital, AP-HP, Paris Centre University, Paris, France.
Laboratory of Onco-Hematology, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.
CNRS, INSERM U1151, Institut Necker Enfants Malades (INEM), Université Paris Cité, Paris, France.
Hematology Laboratory, Assistance Publique-Hôpitaux de Paris, Cochin Hospital, Paris, France.
INSERM U1016, Université de Paris, Institut Cochin, Paris, France.
Rheumatology Department, Competence Center for Rare Genetic Bone Disorders, University-Hospital of Strasbourg, Strasbourg, France.
CEREMAST, the Department of Dermatology, Allergology Unit, University Hospital of Besançon, Besançon, France.
Unité Transversale Allergologie, Dermatologie, CHU Besançon - Université de Franche-Comté, EFS, INSERM, UMR RIGHT, Besançon, France.
Unité de soins et de recherche en médecine interne et maladies infectieuses, European Hospital, Marseille, France.
Department of Haematology, Sud Reunion University Hospital, Saint Pierre, France.
CEREMAST, the Department of Hematology, CHU Dupuytren, Limoges, France.
CEREMAST, the Department of Dermatology, CHU de Poitiers, Poitiers, France.
Department of Internal Medicine, Hôpital Haut-Lévêque, Université de BORDEAUX, Pessac, France.
Department of Internal Medicine, Angers University Hospital, Angers, France.
The Service d'Immunologie Clinique et d'Allergologie, Centre Hospitalier Régional Universitaire, Tours, France.
Internal Medicine Department, Centre Hospitalier Lyon Sud, Pierre-Bénite, France.
INSERM U1290, Université Lyon 1, Lyon, France.
Department of Hematology, CHU de Bordeaux, Bordeaux, France.
Service d'hématologie Oncologique et Thérapie Cellulaire, CIC-1402, CHU de Poitiers, Poitiers, France.
Department of Haematology, Hospital Centre of Roubaix, Roubaix, France.
Department of Dermatology and Allergy, Tenon Hospital, Sorbonne University, Paris, France.
Platform of Molecular Analysis for Mastocytosis and MCAD (CEREMAST), Department of Biological Hematology, Pitié-Salpêtrière Hospital, AP-HP, Paris Sorbonne University, Paris, France.

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.

中文翻译：

根据 WHO 分类对 170 例接受米哚妥林治疗的晚期肥大细胞增多症和 C 发现患者的预后评分比较

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，与不良结局相关。尽管米哚妥林是第一个被批准用于 AdvSM 患者的酪氨酸激酶抑制剂，但长期反应是有限的。已经建立了突变调整风险评分（MARS）、肥大细胞增多症国际预后评分系统（IPSM）和系统性肥大细胞增多症全球预后评分（GPSM）来表征总体 AdvSM 患者的预后。然而，鉴于结果对 AdvSM 亚型的依赖性，每种亚型的预后特征至关重要。我们旨在根据 AdvSM 亚型使用预后评分的 Harrell 相关指数研究预测能力。我们使用法国肥大细胞增多症参考中心的登记处进行了一项全国性的回顾性研究，并纳入了所有接受米哚妥林治疗的 C 发现患者。总共确定了 170 例患者： 46 例侵袭性 SM （ASM）、 11 例肥大细胞白血病（MCL）和 113 例 SM 伴相关血液肿瘤（SM-AHN）。当与 AdvSM 亚型联合时，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 指数 = 0.689），其次是 GPSM （C 指数 = 0.677）和 IPSM （C 指数 = 0.618）。在多变量分析中，MARS 分层和 AdvSM 亚型均预后为 OS。因此，确定了 5 个具有不同中位 OS 的 AdvSM 患者亚组： MCL 为 9.9 个月，中/高危 SM-AHN 为 24 个月，中/高危 ASM 为 33 个月，低危 SM-AHN 为 58 个月，低危 ASM 未达到（p < 0.001）。 AdvSM 亚型和 MARS 对 OS 的预测性最强，应提示进行特定管理。

更新日期：2024-09-17

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