ArticleMaedica2025
Innovative Therapeutic Approaches in Systemic Mastocytosis: an Updated Review.
Article in Maedica, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Introduction: Systemic mastocytosis (SM) is a heterogeneous clonal disorder characterized by the accumulation of abnormal mast cells in various tissues, predominantly the bone marrow. Given the rarity of the disease, the available data in Romania are extremely limited. It is estimated that the total number of diagnosed patients is approximately 170. In recent years, significant advances have been made in understanding the molecular pathogenesis of SM, leading to the development of targeted therapies that have transformed the management of this condition. The approval of tyrosine kinase inhibitors (TKIs), particularly midostaurin and avapritinib, has provided new therapeutic options for patients with advanced SM, demonstrating significant improvements in overall survival (OS) and symptom control. Objective: To conduct a systematic review of clinical trials, observational studies and international or national guidelines published since 2000 to evaluate the efficacy, safety and mechanistic rationale of innovative therapeutic approaches for SM - including KIT inhibitors, monoclonal antibody-based therapies, stem cell transplant - and to compare the impact of these interventions with conventional cytoreductive and symptomatic treatments on overall response rate, survival, mediator-related symptom burden, quality of life and treatment-related adverse events. Materials and methods: A comprehensive search was performed in MEDLINE ( via PubMed), Embase, the Cochrane Central Register of Controlled Trials and Web of Science from 1 January 2000 to 1 May 2025. ClinicalTrials.gov, WHO ICTRP. Search strings combined controlled vocabulary ( e.g. , "Mastocytosis") and free-text terms for the disease (systemic mastocytosis, advanced SM, indolent SM) with key innovative interventions (KIT inhibitors, avapritinib, midostaurin, monoclonal antibodies, stem cell transplant). No language limits were set at the search stage; non-English full texts were excluded only if an accurate translation could not be obtained. As the review relied exclusively on published or publicly available data, ethical approval and informed consent were not required. Conclusion: The present review provides an updated overview of the evolving therapeutic landscape of SM, emphasizing recent clinical trial data, novel targeted therapies and emerging treatment paradigms. We discuss the implications of this progress on patient outcomes and future directions for personalized medicine in SM.
Identifiers
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.