Midostaurin improves quality of life and mediator-related symptoms in advanced systemic mastocytosis.,Journal of Allergy and Clinical Immunology

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Midostaurin improves quality of life and mediator-related symptoms in advanced systemic mastocytosis.
Journal of Allergy and Clinical Immunology ( IF 11.4 ) Pub Date : 2020-05-11 , DOI: 10.1016/j.jaci.2020.03.044
Karin Hartmann ₁ , Jason Gotlib ₂ , Cem Akin ₃ , Olivier Hermine ₄ , Farrukh T Awan ₅ , Elizabeth Hexner ₆ , Michael J Mauro ₇ , Hans D Menssen ₈ , Suman Redhu ₈ , Stefanie Knoll ₉ , Karl Sotlar ₁₀ , Tracy I George ₁₁ , Hans-Peter Horny ₁₂ , Peter Valent ₁₃ , Andreas Reiter ₁₄ , Hanneke C Kluin-Nelemans ₁₅

Affiliation

Division of Allergy, Department of Dermatology, University of Basel, Basel, Switzerland; Department of Biomedicine, University of Basel, Basel, Switzerland.
Stanford University School of Medicine, Stanford, Calif.
Brigham and Women's Hospital, Boston, Mass.
University of Paris Descartes, Institut Imagine, Centre de Reference des Mastocytoses, Paris, France.
Harold C. Simmons Comprehensive Cancer Center University of Texas Southwestern Medical Center, Dallas, Tex.
University of Pennsylvania, Philadelphia, Pa.
Memorial Sloan Kettering Cancer Center, New York, NY.
Novartis Pharma AG, Basel, Switzerland.
Novartis Pharmaceuticals Corporation, East Hanover, NJ.
Institute of Pathology, Paracelsus Medical University Salzburg, Austria.
University of Utah and ARUP Laboratories, Salt Lake City, Utah.
Ludwig-Maximilians-University Munich, Munich, Germany.
Department of Internal Medicine I, Division of Hematology and Hemostaseology and Ludwig Boltzmann Cluster Oncology, Medical University of Vienna, Vienna, Austria.
Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University, Mannheim, Germany.
University Medical Center Groningen, Department of Hematology, University of Groningen, Groningen, The Netherlands.

Background

Advanced systemic mastocytosis (advSM) is characterized by presence of the KIT D816V mutation and pathologic accumulation of neoplastic mast cells (MCs) in various tissues, leading to severe symptoms and organ damage (eg, cytopenias, liver dysfunction, portal hypertension, malabsorption, and weight loss). Treatment with midostaurin, an orally active multikinase/KIT inhibitor now approved for advSM in the United States and the European Union, resulted in a high rate of response accompanied by reduced MC infiltration of the bone marrow and lowered serum tryptase level.

Objective

We aimed to determine whether midostaurin improves health-related quality of life (QOL) and MC mediator–related symptoms in patients with advSM.

Methods

In 116 patients with systemic mastocytosis (89 patients with advSM fulfilling the strict inclusion criteria of the D2201 study [ClinicalTrials.gov identifier NCT00782067]), QOL and symptom burden were assessed during treatment with midostaurin by using the 12-Item Short-Form Health Survey (SF-12) and the Memorial Symptom Assessment Scale patient-reported questionnaires, respectively. MC mediator–related symptoms were evaluated by using a specific physician-reported questionnaire.

Results

Over the first 6 cycles of treatment with midostaurin (ie, 6 months), patients experienced significant improvements in total SF-12 and Memorial Symptom Assessment Scale scores, as well as in subscores of each instrument. These improvements were durable during 36 months of follow-up. Similarly, we found substantial improvements (67%-100%) in all MC mediator–related symptoms.

Conclusion

QOL and MC mediator–related symptoms significantly improve with midostaurin treatment in patients with advSM (ClinicalTrials.gov identifier, NCT00782067).

中文翻译：

Midostaurin可改善晚期全身性肥大细胞增多症的生活质量和与介体有关的症状。

背景

晚期全身肥大细胞增多症（advSM）的特征是在各种组织中都存在KIT D816V突变和肿瘤性肥大细胞（MCs）的病理积累，从而导致严重的症状和器官损害（例如，血细胞减少，肝功能不全，门脉高压，吸收不良和减肥）。Midostaurin是一种口服活性多激酶/ KIT抑制剂，目前已在美国和欧盟批准用于advSM的治疗，导致反应率高，同时骨髓MC浸润减少，血清类胰蛋白酶水平降低。

目的

我们旨在确定Midostaurin是否能改善advSM患者的健康相关生活质量（QOL）和MC介体相关症状。

方法

在116例系统性肥大细胞增多症患者中（89例advSM患者符合D2201研究的严格纳入标准[ClinicalTrials.gov标识符NCT00782067]），在米ostaurin治疗期间通过使用12项简短形式健康调查评估了生活质量和症状负担（SF-12）和纪念症状评估量表的患者报告问卷。通过使用医生报告的特定问卷评估与MC介体有关的症状。