Abstract
The American Initiative in Mast Cell Diseases (AIM) held its inaugural investigator conference at Stanford University School of Medicine in May 2019. The overarching goal of this meeting was to establish a Pan-American organization of physicians and scientists with multidisciplinary expertise in mast cell disease. To serve this unmet need, AIM envisions a network where basic, translational, and clinical researchers could establish collaborations with both academia and biopharma to support the development of new diagnostic methods, enhanced understanding of the biology of mast cells in human health and disease, and the testing of novel therapies. In these AIM proceedings, we highlight selected topics relevant to mast cell biology and provide updates regarding the recently described hereditary alpha-tryptasemia. In addition, we discuss the evaluation and treatment of mast cell activation (syndromes), allergy and anaphylaxis in mast cell disorders, and the clinical and biologic heterogeneity of the more indolent forms of mastocytosis. Because mast cell disorders are relatively rare, AIM hopes to achieve a coordination of scientific efforts not only in the Americas but also in Europe by collaborating with the well-established European Competence Network on Mastocytosis.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 2043-2052 |
| Number of pages | 10 |
| Journal | Journal of Allergy and Clinical Immunology |
| Volume | 147 |
| Issue number | 6 |
| DOIs | |
| State | Published - Jun 2021 |
Funding
The research reported in this publication was supported by the National Center for Advancing Translational Sciences of the National Institutes of Health (NIH) (award no. R13TR002722 to J.G.). The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. We thank The Mast Cell Disease Society, Inc (TMS), a national 501c3 nonprofit, for their partnership and support of AIM, for patient-centered research, and for sponsoring international physicians at this inaugural meeting. J.G. expresses gratitude for the support of the Charles and Ann Johnson Foundation, the staff of the Stanford Mastocytosis Center, and the Stanford Cancer Institute Innovation Fund. M.C., J.J.L., and D.D.M. are supported in part by the Division of Intramural Research of the National Institute of Allergy and Infectious Diseases , NIH. D.F.D. is supported by the Asthma and Allergic Diseases Cooperative Research Centers Opportunity Fund (award no. U19AI07053 from the NIH). P.V. has been supported by the Austrian Science Fund (FWF) (grant nos. F4701-B20 , F4704-B20 , and P32470-B ). Disclosure of potential conflict of interest: J. Gotlib has served as a Chair of the Study Steering Committee (SSC) for the global trial of midostaurin in advanced systemic mastocytosis (advSM) (Novartis), Chair of the Response Adjudication Committee (RAC) for studies of avapritinib in advSM (Blueprint Medicines), and SSC co-chair for the phase II trial of ripretinib in advSM (Deciphera Pharmaceuticals); has received funding for the conduct of these trials; and has received honoraria and reimbursement of travel expenses from Novartis, Blueprint Medicines, and Deciphera Pharmaceuticals. T.I. George has served on the SSC for clinical trials in SM for Novartis and Blueprint Medicines and has received consulting fees and reimbursement of travel expenses from Novartis and Blueprint Medicines. B. Bochner receives remuneration for serving on the Scientific Advisory Board of Allakos, Inc; owns stock in Allakos; receives publication-related royalty payments from Elsevier and UpToDate; is a coinventor on existing Siglec-8–related patents and thus may be entitled to a share of royalties received by Johns Hopkins University during development and potential sales of such products; and is also a cofounder of Allakos, which makes him subject to certain restrictions under University policy. The terms of this arrangement are being managed by Johns Hopkins University and Northwestern University in accordance with their conflict-of-interest policies. M. J. Hamilton serves on a Scientific Advisory Board for Allakos. L. B. Schwartz is a consultant for Deciphera Pharmaceuticals, Blueprint Medicines, Allakos, and Genentech; participated in clinical trials sponsored by Deciphera Pharmaceuticals and Blueprint Medicines; and, as inventor of the commercial tryptase assay, receives funds from VCU collected as royalties from Thermo Fisher. C. Ustun has served as a consultant and received honoraria from Incyte, Inc, and Jazz Pharmaceuticals. H.-P. Horny serves on a scientific advisory board and SSC for Blueprint Medicines and Deciphera Pharmaceuticals; and has served as a consultant and received honoraria from Blueprint Medicines, Deciphera Pharmaceuticals, and Novartis. A. Orfao has received consultancy honoraria from Novartis. M. Deininger is a paid consultant and/or member of the Scientific Advisory Board for Fusion Pharma, Takeda, Novartis, Incyte, Sangama, SPARC, Pfizer, and DisperSol; and serves on the SSC for the Optic (Takeda), EXPLORER (Blueprint), PATHFINDER (Blueprint), and BFORE (Pfizer) clinical trials. D. Radia received funding for the conduct of the global trial of midostaurin in advSM (Novartis), and for studies of avapritinib in advSM (Blueprint Medicines); and has also served on the RAC for studies of avapritinib in advSM (Blueprint Medicines). M. Jawhar received consultancy honoraria from Novartis and Blueprint, and research support from Novartis. H. Kluin-Nelemans received institutional support from Novartis. M. Arock has served as a consultant and received honoraria from Blueprint Medicines and Novartis and has received a research grant from Deciphera Pharmaceuticals. W. R. Sperr received honoraria from Thermo Fisher, AbbVie, Novartis, Pfizer, Incyte, Deciphera Pharmaceuticals, Jazz Pharmaceuticals, Teva, and Celgene. P. Valent received consultancy honoraria from Novartis, Incyte, Blueprint, Deciphera Pharmaceuticals, and Thermo Fisher, and research support from Novartis, Blueprint, and Deciphera Pharmaceuticals. M. Castells is a consultant for Blueprint Medicines and one of the PIs of the PIONEER clinical trial for indolent SM. C. Akin has received research support from Blueprint Medicines and has served as a consultant for Blueprint Medicines and Novartis, including SSC for clinical trials in SM. The rest of the authors declare that they have no relevant conflicts of interest. The research reported in this publication was supported by the National Center for Advancing Translational Sciences of the National Institutes of Health (NIH) (award no. R13TR002722 to J.G.). The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. We thank The Mast Cell Disease Society, Inc (TMS), a national 501c3 nonprofit, for their partnership and support of AIM, for patient-centered research, and for sponsoring international physicians at this inaugural meeting. J.G. expresses gratitude for the support of the Charles and Ann Johnson Foundation, the staff of the Stanford Mastocytosis Center, and the Stanford Cancer Institute Innovation Fund. M.C., J.J.L., and D.D.M. are supported in part by the Division of Intramural Research of the National Institute of Allergy and Infectious Diseases, NIH. D.F.D. is supported by the Asthma and Allergic Diseases Cooperative Research Centers Opportunity Fund (award no. U19AI07053 from the NIH). P.V. has been supported by the Austrian Science Fund (FWF) (grant nos. F4701-B20, F4704-B20, and P32470-B).
Keywords
- American Initiative in Mast Cell Diseases (AIM)
- European Competence Network on Mastocytosis (ECNM)
- Systemic mastocytosis
- anaphylaxis
- hereditary alpha-tryptasemia
- mast cell activation syndrome
- siglec-8
ASJC Scopus subject areas
- Immunology and Allergy
- Immunology
