Refined Treatment Response Criteria for Indolent Systemic Mastocytosis Proposed by the ECNM-AIM Consortium
Open accessAbstract
Indolent systemic mastocytosis (ISM) has a favorable prognosis and normal life expectancy. However, many patients suffer from mast cell (MC) mediator-related symptoms, which significantly affect quality of life (QoL). Cutaneous, gastrointestinal, and neurological complaints, musculoskeletal pain, and the presence of skin lesions, anaphylaxis, and osteoporosis are the main symptoms and signs in ISM and must be assessed in all patients before and during treatment. Validated mastocytosis-specific patient-reported outcome measures (PROMs) should be used for this purpose. Serum tryptase and KIT D816V allele burden are recommended as secondary outcome parameters, noting that they do not reflect the severity of signs, symptoms, and related QoL impairment, but indirectly express MC burden. Changes from baseline of 90%, 60%, and 30% indicate complete response >90%, major response 60% to 90%, partial response 30% to 60%, and no response <30% to treatment. To conclude, we recommend the use of PROMs as primary outcome parameters to define treatment response in patients with ISM in clinical trials and in everyday clinical practice.
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Tryptase in patients with systemic mastocytosis is not correlated with symptom severity.
“Importantly, tryptase in patients with SM is not correlated with symptom severity [71].”
From Diagnostic Significance of Tryptase for Suspected Mast Cell Disorders (2023) · cites this paper as [71]
Most cases of ISM present a clinical picture dominated by mediator-related symptoms, including cutaneous, gastrointestinal, musculoskeletal, and neurocognitive manifestations.
“Conversely, most cases of ISM present a clinical picture dominated by mediator-related symptoms, including cutaneous, gastrointestinal, musculoskeletal, and neurocognitive manifestations (54–56).”
From Defining “Normal” basal serum tryptase levels: a context-dependent approach to improve diagnostics in systemic mastocytosis (2025) · cites this paper as [55]
In cases with less specific manifestations (gastrointestinal symptoms, fatigue, neurologic/neuropsychiatric manifestations), recommendations for testing BST are not standardized.
“In all these cases, the recommendations for testing BST are not standardized (53–55, 65).”
From Defining “Normal” basal serum tryptase levels: a context-dependent approach to improve diagnostics in systemic mastocytosis (2025) · cites this paper as [55]
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References 82 · Cited by 27
- Authors
- Pyatilova, P., Akin, C., Alvarez-Twose, I., Arock, M., Bonadonna, P., Brockow, K., Butterfield, J. H., Broesby-Olsen, S., Carter, M. C., Castells, M., George, T. I., Gotlib, J., Greiner, G., Gülen, T., Hartmann, K., Hermine, O., Horny, H.-P., Jawhar, M., Lange, M., Lyons, J. J., Maurer, M., Metcalfe, D. D., Nedoszytko, B., Niedoszytko, M., Orfao, A., Reiter, A., Schwaab, J., Sotlar, K., Sperr, W. R., Triggiani, M., Valent, P., & Siebenhaar, F.
- Affiliations
- Humboldt-Universität zu Berlin (DE)
- University of Michigan (US)
- Pitié-Salpêtrière Hospital (FR)
- Azienda Ospedaliera Universitaria Integrata Verona (IT)
- Technical University of Munich (DE)
- Mayo Clinic (US)
- National Institutes of Health (US)
- Brigham and Women's Hospital (US)
- University of Utah (US)
- Stanford Cancer Institute (US)
- Ludwig Boltzmann Institute for Hematology and Oncology (AT)
- Karolinska University Hospital (SE)
- University of Basel (CH)
- Inserm (FR)
- Pathologisches Institut (DE)
- University Medical Centre Mannheim (DE)
- Gdańsk Medical University (PL)
- Paracelsus Medical University (AT)
- University of Salerno (IT)
- Corresponding author
- Siebenhaar, Frank
- ORCID
- Pyatilova, Polina
- Akin, Cem
- Arock, Michel
- Bonadonna, Patrizia
- Brockow, Knut
- Butterfield, Joseph H.
- Carter, Melody C.
- Castells, Mariana
- George, Tracy I.
- Gotlib, Jason
- Greiner, Georg
- Gülen, Theo
- Hartmann, Karin
- Hermine, Olivier
- Lange, Magdalena
- Lyons, Jonathan J.
- Maurer, Marcus
- Metcalfe, Dean D.
- Niedoszytko, Marek
- Reiter, Andreas
- Schwaab, Juliana
- Sotlar, Karl
- Sperr, Wolfgang R.
- Triggiani, Massimo
- Valent, Peter
- Siebenhaar, Frank
- Venue
- The Journal of Allergy and Clinical Immunology In Practice
- Publisher
- Elsevier BV
- Published
- 2022-08
- Volume
- 10
- Issue
- 8
- Pages
- 2015-2024
- ISSN
- 2213-2198 (ISSN-L)
- OpenAlex
- W4283068008
- Cited by
- 27
- References
- 82
- Citations by year
- 2026: 1 · 2025: 7 · 2024: 7 · 2023: 9 · 2022: 3
- FWCI
- 2.48
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- The Journal of Allergy and Clinical Immunology In Practice (acceptedVersion)
- PubMed (publishedVersion)
- University of Southern Denmark Research Portal (University of Southern Denmark) (publishedVersion)
- DIGITAL.CSIC (Spanish National Research Council (CSIC)) (submittedVersion)
- mediaTUM (Technical University of Munich) (submittedVersion)
- Fraunhofer-Publica (Fraunhofer-Gesellschaft) (submittedVersion)
- Topics
- Life Sciences › Immunology and Microbiology › Immunology › Mast cells and histamine
- Health Sciences › Medicine › Gastroenterology › Gastrointestinal Tumor Research and Treatment
- Health Sciences › Medicine › Rheumatology › Eosinophilic Disorders and Syndromes
- Keywords
- indolent systemic mastocytosis, treatment response criteria, patient-reported outcome measures, quality of life, serum tryptase, complete response, clinical trial endpoints
- MeSH terms
- Humans, Mast Cells, Mastocytosis, Quality of Life, Mastocytosis, Systemic, Tryptases
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