Systemic mastocytosis
Carboplatin & Paclitaxel (CP)
CP: Carboplatin & Paclitaxel
Regimen
Study | Dates of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Forde et al. 2022 (CheckMate 816) | 2017-2019 | Phase 3 (C) | 1a. CP & Nivolumab 1b. CVb & Nivolumab 1c. DC & Nivolumab |
Inferior EFS |
Note: there were additional comparator options depending on histology; see the respective histology-specific pages for more details. This study was conducted in the United States. The reason for the study was that an unanswered question at the time was whether adding an immune checkpoint inhibitor would improve outcomes.
Biomarker eligibility criteria
- CheckMate 816: No sensitizing EGFR or ALK mutations
Chemotherapy
- Carboplatin (Paraplatin) AUC 5 or 6 IV once on day 1
- Paclitaxel (Taxol) 175 or 200 mg/m2 IV once on day 1
21-day cycle for 3 cycles
Subsequent treatment
References
- CheckMate 816: Forde PM, Spicer J, Lu S, Provencio M, Mitsudomi T, Awad MM, Felip E, Broderick SR, Brahmer JR, Swanson SJ, Kerr K, Wang C, Ciuleanu TE, Saylors GB, Tanaka F, Ito H, Chen KN, Liberman M, Vokes EE, Taube JM, Dorange C, Cai J, Fiore J, Jarkowski A, Balli D, Sausen M, Pandya D, Calvet CY, Girard N; CheckMate 816 Investigators. Neoadjuvant Nivolumab plus Chemotherapy in Resectable Lung Cancer. N Engl J Med. 2022 May 26;386(21):1973-1985. Epub 2022 Apr 11. link to original article contains dosing details in manuscript PubMed NCT02998528
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Section editor | |
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Sanjay R. Mohan, MD, MSCI Nashville, TN |
7 regimens on this page
13 variants on this page
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Note: most published trials in this area are for "advanced systemic mastocytosis", which includes more than one WHO category. See individual regimens for details.
All lines of therapy
Allogeneic hematopoietic cell transplant
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To our knowledge, only retrospective analyses have been published.
References
- Retrospective: Ustun C, Reiter A, Scott BL, Nakamura R, Damaj G, Kreil S, Shanley R, Hogan WJ, Perales MA, Shore T, Baurmann H, Stuart R, Gruhn B, Doubek M, Hsu JW, Tholouli E, Gromke T, Godley LA, Pagano L, Gilman A, Wagner EM, Shwayder T, Bornhäuser M, Papadopoulos EB, Böhm A, Vercellotti G, Van Lint MT, Schmid C, Rabitsch W, Pullarkat V, Legrand F, Yakoub-Agha I, Saber W, Barrett J, Hermine O, Hagglund H, Sperr WR, Popat U, Alyea EP, Devine S, Deeg HJ, Weisdorf D, Akin C, Valent P. Hematopoietic stem-cell transplantation for advanced systemic mastocytosis. J Clin Oncol. 2014 Oct 10;32(29):3264-74. Epub 2014 Aug 25. link to original article link to PMC article PubMed
Cladribine monotherapy
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Regimen #1
Study | Evidence |
---|---|
Kluin-Nelemans et al. 2003 | Pilot, <20 patients reported |
Chemotherapy
- Cladribine (Leustatin) 0.13 mg/kg IV over 2 hours once per day on days 1 to 5
Supportive medications
- Clemastine (Tavist) 1 to 2 mg IV prior to Cladribine (Leustatin)
- Dexamethasone (Decadron) 5 mg IV prior to Cladribine (Leustatin)
- Fluconazole (Diflucan) (dose not specified)
- Co-trimaxazole (dose not specified)
- Valacyclovir (Valtrex) (dose not specified) "in some patients"
4 to 6-week cycle for 6 cycles
Regimen #2
Study | Evidence |
---|---|
Lim et al. 2009 | Retrospective |
This is a retrospective series.
Chemotherapy
- Cladribine (Leustatin) 5 mg/m2 or 0.13 to 0.17 mg/kg IV over 2 hours once per day on days 1 to 5
Median number of treatment cycles was 3 (range 1 to 9)
Regimen #3
Study | Evidence |
---|---|
Hermine et al. 2010 | Retrospective |
Chemotherapy
- Cladribine (Leustatin) 0.15 mg/kg IV over 2 hours or SC once per day on days 1 to 5
4 to 12-week cycle for 1 to 6 cycles
References
- Kluin-Nelemans HC, Oldhoff JM, Van Doormaal JJ, Van 't Wout JW, Verhoef G, Gerrits WB, van Dobbenburgh OA, Pasmans SG, Fijnheer R. Cladribine therapy for systemic mastocytosis. Blood. 2003 Dec 15;102(13):4270-6. Epub 2003 Aug 21. link to original article contains verified protocol PubMed
- Retrospective: Lim KH, Pardanani A, Butterfield JH, Li CY, Tefferi A. Cytoreductive therapy in 108 adults with systemic mastocytosis: Outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine. Am J Hematol. 2009 Dec;84(12):790-4. link to original article PubMed
- Abstract: Retrospective: Hermine, Olivier, Hirsh, Isabelle, Damaj, Gandhi, Granpeix, Catherine, Barete, Stephane, Suarez, Felipe, Chansderis, Olivia, Ghez, David, Delarue, Richard, Lanternier, Fanny, Deau, Benedicte, Coignard, Helene, Fraitag, Sylvie, Canioni, Danielle, Casassus, Philippe, Dubreuil, Patrice, Lortholary, Olivier. Long Term Efficacy and Safety of Cladribine In Adult Systemic mastocytosis: a French Multicenter Study of 44 Patients. ASH Annual Meeting Abstracts 2010 116: 1982 link to original abstract
- Update: Retrospective: Barete S, Lortholary O, Damaj G, Hirsch I, Chandesris MO, Elie C, Hamidou M, Durieu I, Suarez F, Grosbois B, Limal N, Gyan E, Larroche C, Guillet G, Kahn JE, Casassus P, Amazzough K, Coignard-Biehler H, Georgin-Lavialle S, Lhermitte L, Fraitag S, Canioni D, Dubreuil P, Hermine O. Long-term efficacy and safety of cladribine (2-CdA) in adult patients with mastocytosis. Blood. 2015 Aug 20;126(8):1009-16; quiz 1050. Epub 2015 May 22. link to original article PubMed
Dasatinib monotherapy
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Regimen
Study | Evidence |
---|---|
Verstovsek et al. 2008 | Phase II |
Only 9 of 67 patients studied had ASM; 18 has ISM and 6 had SM-AHNMD.
Chemotherapy
- Dasatinib (Sprycel) 70 mg PO BID or 140 mg PO once per day
Supportive medications
- Hematopoietic growth factors at investigator discretion
28-day cycles until disease progression or intolerable toxicity
References
- Verstovsek S, Tefferi A, Cortes J, O'Brien S, Garcia-Manero G, Pardanani A, Akin C, Faderl S, Manshouri T, Thomas D, Kantarjian H. Phase II study of dasatinib in Philadelphia chromosome-negative acute and chronic myeloid diseases, including systemic mastocytosis. Clin Cancer Res. 2008 Jun 15;14(12):3906-15. link to original article contains verified protocol link to PMC article PubMed
Imatinib monotherapy
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Regimen #1
Study | Evidence |
---|---|
Vega-Ruiz et al. 2009 | Phase II |
Chemotherapy
- Imatinib (Gleevec) 400 mg PO once per day, increased by 200 mg every month to a maximum of 800 mg PO once per day
Supportive medications
- Hematopoietic growth factors at investigator discretion
Continued until disease progression or unacceptable toxicity.
Regimen #2
Study | Evidence |
---|---|
Droogendijk et al. 2006 | Phase II, <20 pts reported |
Chemotherapy
- Imatinib (Gleevec) 400 mg PO once per day
Supportive medications
- Prednisone (Sterapred) 15 mg PO BID for first 2 weeks of treatment
Up to 6 month course
References
- Pardanani A, Elliott M, Reeder T, Li CY, Baxter EJ, Cross NC, Tefferi A. Imatinib for systemic mast-cell disease. Lancet. 2003 Aug 16;362(9383):535-6. link to original article PubMed
- Droogendijk HJ, Kluin-Nelemans HJ, van Doormaal JJ, Oranje AP, van de Loosdrecht AA, van Daele PL. Imatinib mesylate in the treatment of systemic mastocytosis: a phase II trial. Cancer. 2006 Jul 15;107(2):345-51. link to original article contains verified protocol PubMed
- Retrospective: Pagano L, Valentini CG, Caira M, Rondoni M, Van Lint MT, Candoni A, Allione B, Cattaneo C, Marbello L, Caramatti C, Pogliani EM, Iannitto E, Giona F, Ferrara F, Invernizzi R, Fanci R, Lunghi M, Fianchi L, Sanpaolo G, Stefani PM, Pulsoni A, Martinelli G, Leone G, Musto P. Advanced mast cell disease: an Italian Hematological Multicenter experience. Int J Hematol. 2008 Dec;88(5):483-8. Epub 2008 Nov 26. link to original article PubMed
- Vega-Ruiz A, Cortes JE, Sever M, Manshouri T, Quintás-Cardama A, Luthra R, Kantarjian HM, Verstovsek S. Phase II study of imatinib mesylate as therapy for patients with systemic mastocytosis. Leuk Res. 2009 Nov;33(11):1481-4. Epub 2009 Feb 3. link to original article contains verified protocol link to PMC article PubMed
- Retrospective: Lim KH, Pardanani A, Butterfield JH, Li CY, Tefferi A. Cytoreductive therapy in 108 adults with systemic mastocytosis: Outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine. Am J Hematol. 2009 Dec;84(12):790-4. link to original article PubMed
Interferon alfa-2b monotherapy
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Regimen
Study | Evidence |
---|---|
Casassus et al. 2002 | Phase II |
Doses were escalated based on tolerance.
Chemotherapy
- Interferon alfa-2b (Intron-A) as follows:
- Week 1: 1 million units (MU) SC once per day
- Week 2: 3 MU SC once per day
- Weeks 3 & 4: 3 MU/m2 SC once per day
- Week 5 onwards: 5 MU/m2 SC once per day
6-month course, continued at physician's and patient's discretion
References
- Casassus P, Caillat-Vigneron N, Martin A, Simon J, Gallais V, Beaudry P, Eclache V, Laroche L, Lortholary P, Raphaël M, Guillevin L, Lortholary O. Treatment of adult systemic mastocytosis with interferon-alpha: results of a multicentre phase II trial on 20 patients. Br J Haematol. 2002 Dec;119(4):1090-7. link to original article contains verified protocol PubMed
- Retrospective: Lim KH, Pardanani A, Butterfield JH, Li CY, Tefferi A. Cytoreductive therapy in 108 adults with systemic mastocytosis: Outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine. Am J Hematol. 2009 Dec;84(12):790-4. link to original article PubMed
Midostaurin monotherapy
Midostaurin (Rydapt) for aggressive systemic mastocytosis
Nilotinib monotherapy
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Regimen
Study | Evidence |
---|---|
Hochhaus et al. 2015 | Phase II |
Chemotherapy
- Nilotinib (Tasigna) 400 mg PO BID
28-day cycles until disease progression or unacceptable toxicity
References
- Hochhaus A, Baccarani M, Giles FJ, le Coutre PD, Müller MC, Reiter A, Santanastasio H, Leung M, Novick S, Kantarjian HM. Nilotinib in patients with systemic mastocytosis: analysis of the phase 2, open-label, single-arm nilotinib registration study. J Cancer Res Clin Oncol. 2015 Nov;141(11):2047-60. Epub 2015 May 23. contains verified protocol link to PMC article PubMed
Response criteria
IWG-MRT & ECNM consensus response criteria (2013)
- Gotlib J, Pardanani A, Akin C, Reiter A, George T, Hermine O, Kluin-Nelemans H, Hartmann K, Sperr WR, Brockow K, Schwartz LB, Orfao A, Deangelo DJ, Arock M, Sotlar K, Horny HP, Metcalfe DD, Escribano L, Verstovsek S, Tefferi A, Valent P. International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) & European Competence Network on Mastocytosis (ECNM) consensus response criteria in advanced systemic mastocytosis. Blood. 2013 Mar 28;121(13):2393-401. Epub 2013 Jan 16. link to original article link to PMC article PubMed