Systemic mastocytosis

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7 regimens on this page 13 variants on this page

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

1. 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

1. 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
2. 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
3. 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
   1. 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

1. 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

1. 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
2. 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
3. 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
4. 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
5. 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

1. 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
2. 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

1. 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)

1. 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