Medication Use in Indolent Systemic Mastocytosis (ISM) in Germany Prior to Avapritinib EMA Approval
Author(s)
Marcel Herold, MA1, Hedwig Silies, MPH, PhD1, Svitlana Schnaidt, MsC2, Christian Jacob, PhD2.
1Blueprint Medicines (Germany) GmbH, München, Germany, 2Cencora, Hannover, Germany.
1Blueprint Medicines (Germany) GmbH, München, Germany, 2Cencora, Hannover, Germany.
OBJECTIVES: Systemic mastocytosis (SM) is a clonal mast cell disease associated with the KIT D816V mutation and caused by mast cell accumulation and activation. SM patients are characterized by debilitating heterogenous manifestations, including anaphylaxis. This study aimed to assess the medications used in ISM prior to the EMA approval of avapritinib in December 2023.
METHODS: A retrospective analysis based on German claims data from the InGef research database was conducted. For the analysis of ISM medication use for 2021, a subset of adult patients with at least two ICD‑10‑GM diagnosis codes for ISM (D47.0) in two different quarters in the inpatient and/or outpatient sector were selected. Assessed medications (based on the Anatomical Therapeutic Chemical (ATC) and German procedure (OPS) classification codes, if applicable) were predefined and included the treatment options for SM according to the German SM Onkopedia guidelines.
RESULTS: In 2021, 77% of the ISM population received at least one treatment. Most used treatments were best supportive care (BSC) like H2 antihistamines (50%), H1 antihistamines (46%), corticosteroids (44%), adrenaline auto-injectors (22%), and proton pump inhibitors (14%). Omalizumab was rarely used (less than 3.3% in all cases). Cytoreductive treatments such as cladribine and interferon-alpha or peginterferon alfa-2a were not used at all in this subset. Other treatment options that can be used in the treatment of advanced SM but are not approved for use in ISM were rarely employed, e.g. midostaurin (less than 3.3%), cladribine (0%) and interferon (0%)."
CONCLUSIONS: Prior to EMA approval of avapritinib, most ISM patients in Germany were treated with BSC consisting of H1 and H2 antihistamines, corticosteroids, and proton pump inhibitors amongst others. Cytoreductive and targeted off-label therapies such as cladribine and interferon were not utilized in ISM patients. Midostaurin was administered in a few individual cases (less than 3.3%), but it was not considered a standard treatment option.
METHODS: A retrospective analysis based on German claims data from the InGef research database was conducted. For the analysis of ISM medication use for 2021, a subset of adult patients with at least two ICD‑10‑GM diagnosis codes for ISM (D47.0) in two different quarters in the inpatient and/or outpatient sector were selected. Assessed medications (based on the Anatomical Therapeutic Chemical (ATC) and German procedure (OPS) classification codes, if applicable) were predefined and included the treatment options for SM according to the German SM Onkopedia guidelines.
RESULTS: In 2021, 77% of the ISM population received at least one treatment. Most used treatments were best supportive care (BSC) like H2 antihistamines (50%), H1 antihistamines (46%), corticosteroids (44%), adrenaline auto-injectors (22%), and proton pump inhibitors (14%). Omalizumab was rarely used (less than 3.3% in all cases). Cytoreductive treatments such as cladribine and interferon-alpha or peginterferon alfa-2a were not used at all in this subset. Other treatment options that can be used in the treatment of advanced SM but are not approved for use in ISM were rarely employed, e.g. midostaurin (less than 3.3%), cladribine (0%) and interferon (0%)."
CONCLUSIONS: Prior to EMA approval of avapritinib, most ISM patients in Germany were treated with BSC consisting of H1 and H2 antihistamines, corticosteroids, and proton pump inhibitors amongst others. Cytoreductive and targeted off-label therapies such as cladribine and interferon were not utilized in ISM patients. Midostaurin was administered in a few individual cases (less than 3.3%), but it was not considered a standard treatment option.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
RWD123
Topic
Real World Data & Information Systems
Topic Subcategory
Health & Insurance Records Systems
Disease
Oncology, Rare & Orphan Diseases