CLINICAL HETEROGENEITY IN INDOLENT SYSTEMIC MASTOCYTOSIS: A GERMAN CLAIMS DATA ANALYSIS SUPPORTING THE NEED FOR MULTIDISCIPLINARY CARE

Author(s)

Kristina Dittrich, PhD1, Hedwig Silies, MPH, PhD1, Kim Maren Schneider, M.A.2, Svitlana Schnaidt, M.Sc.2, Christian Jacob, PhD2.
1Blueprint Medicines (Germany) GmbH, a Sanofi Company, Munich, Germany, 2Cencora, Hannover, Germany.
OBJECTIVES: Systemic mastocytosis (SM) is a clonal mast cell disorder, primarily driven by KIT D816V mutation in ~95% of cases. Indolent systemic mastocytosis (ISM) represents ~85% of all cases of SM. Multi-organ mast cell infiltration and mediator release leads to heterogenous, non-specific symptoms like cutaneous, gastrointestinal, neurocognitive manifestations, or occasionally life-threatening anaphylaxis. This analysis aims to describe comorbidities, diagnosing physician specialties, and medications in German ISM patients in 2024.
METHODS: A retrospective German claims data analysis utilizing the InGef research database was conducted. Adult ISM patients were identified by ICD-10-GM code D47.0 in two different quarters in the inpatient and/or outpatient sector in 2024. Pre-defined outpatient medication and physician specialties were assessed. Additionally, pre-defined comorbidities (via ICD-10-GM) were assessed for prevalent ISM patients in 2024 and incident ISM patients prior to first ISM diagnosis (2019-2023).
RESULTS: ISM diagnoses were mainly recorded by general practitioners (66.1%), hematologists/oncologists (12.9%), and dermatologists (5.2%). Among 17 different pre-defined diagnoses recorded prior to ISM diagnosis, “headache” (41.5%), “depressive episodes”, “adverse events not elsewhere classified”, and “sleep disorders” (39.6% each) were most frequent. Further, 15 different concurrent pre-defined diagnoses were recorded in prevalent ISM patients, with “congenital mastocytosis” (27.9%), “depressive episodes” (23.4%), and “adverse events not elsewhere classified” (20.7%) as the most common. Overall, 74.2% of ISM patients received at least one medication; most common were antihistamines (H1: 50.6%; H2: 45.5%), corticosteroids (37.3%), and self-injectable epinephrine (24.5%). Avapritinib and omalizumab were each prescribed to 3.0% of ISM patients, while midostaurin was rare (≤2.3%).
CONCLUSIONS: This German real-world analysis highlights the substantial clinical heterogeneity and multi-system disease burden associated with ISM. Our findings support the importance of a multidisciplinary care approach to address the complex disease manifestations. Greater awareness across medical specialties and more coordinated care strategies may improve diagnostic pathways and patient management, ultimately enhancing outcomes.

Conference/Value in Health Info

2026-11, ISPOR Europe 2026, Vienna, Austria

Value in Health, Volume 29, Issue 12S

Code

RWD28

Topic

Epidemiology & Public Health, Patient-Centered Research, Real World Data & Information Systems

Topic Subcategory

Health & Insurance Records Systems

Disease

Oncology, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)

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