Consensus Opinion on Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis

Biology of Blood and Marrow Transplantation

Volumn 22, Issue 8, 2016, Pages 1348-1356

  Ustun, Celalettin ^a   Gotlib, Jason ^b   Popat, Uday ^c   Artz, Andrew ^d   Litzow, Mark ^e   Reiter, Andreas ^f   Nakamura, Ryotaro ^g   Kluin Nelemans, Hanneke C ^h   Verstovsek, Srdan ^c   Gajewski, James ⁱ   Perales, Miguel Angel ^j   George, Tracy ^k   Shore, Tsiporah ^l   Sperr, Wolfgang ^m   Saber, Wael ⁿ   Kota, Vamsi ^o   Yavuz, Akif Selim ^p   Pullarkat, Vinod ^g   Rogosheske, John ^a   Hogan, William ^e   Van Besien, Koen ^l   Hagglund, Hans ^q   Damaj, Gandhi ^r   Arock, Michel ^s   Horny, Hans Peter ^t   Metcalfe, Dean D ^u   Deeg, H Joachim ^v   Devine, Steven ^w   Weisdorf, Daniel ^a   Akin, Cem ^x   Valent, Peter ^m   more..

^a UNIVERSITY OF MINNESOTA   (United States)

^b STANFORD UNIVERSITY   (United States)

^c UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^d UNIVERSITY OF CHICAGO   (United States)

^e MAYO CLINIC   (United States)

^f UNIVERSITY OF HEIDELBERG   (Germany)

^g CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

^h UNIVERSITY MEDICAL CENTER GRONINGEN   (Netherlands)

ⁱ OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^j MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^k UNIVERSITY OF NEW MEXICO SCHOOL OF MEDICINE   (United States)

^l WEILL CORNELL MEDICAL COLLEGE   (United States)

^m MEDICAL UNIVERSITY OF VIENNA   (Austria)

ⁿ MEDICAL COLLEGE OF WISCONSIN   (United States)

^o EMORY UNIVERSITY   (United States)

^p ISTANBUL UNIVERSITY   (Turkey)

^q UPPSALA UNIVERSITY   (Sweden)

^r CAEN UNIVERSITY HOSPITAL   (France)

^s PITIÉ SALPÊTRIÈRE HOSPITAL   (France)

^t UNIVERSITY OF MUNICH   (Germany)

^u NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^v UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^w OHIO STATE UNIVERSITY   (United States)

^x BRIGHAM AND WOMEN S HOSPITAL   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CLADRIBINE; MIDOSTAURIN;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ANAPHYLAXIS; BONE MARROW BIOPSY; CHRONIC LYMPHATIC LEUKEMIA; CHRONIC MYELOMONOCYTIC LEUKEMIA; COMORBIDITY; CONSENSUS; DIARRHEA; DONOR; DONOR LYMPHOCYTE INFUSION; EOSINOPHILIC LEUKEMIA; GASTROINTESTINAL SYMPTOM; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HUMAN; INTERNATIONAL PROGNOSTIC SCORING SYSTEM; INTESTINE INFECTION; LONG TERM SURVIVAL; MYELOABLATIVE CONDITIONING; NOTE; OSTEOPENIA; OSTEOPOROSIS; OSTEOSCLEROSIS; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; PORTAL HYPERTENSION; REDUCED INTENSITY CONDITIONING; SYSTEMIC MASTOCYTOSIS; VOMITING; ALLOTRANSPLANTATION; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; MALE; MASTOCYTOSIS; PATHOLOGY; PROCEDURES; TRANSPLANTATION CONDITIONING;

CONSENSUS; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MALE; MASTOCYTOSIS; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS;

EID: 84981311900     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2016.04.018     Document Type: Note

References (83)

1. 1 Horny, H.P., Valent, P., Histopathological and immunohistochemical aspects of mastocytosis. Int Arch Allergy Immunol 127 (2002), 115–117.
2. 2 Valent, P., Horny, H.P., Escribano, L., et al. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leuk Res 25 (2001), 603–625.
3. 3 Theoharides, T.C., Valent, P., Akin, C., Mast cells, mastocytosis, and related disorders. N Engl J Med 373 (2015), 1885–1886.
4. 4 Arber, D.A., Orazi, A., Hasserjian, R., et al. The 2016 revision to the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia. Blood 127 (2016), 2391–2405.
5. 5 Lim, K.H., Tefferi, A., Lasho, T.L., et al. Systemic mastocytosis in 342 consecutive adults: survival studies and prognostic factors. Blood 113 (2009), 5727–5736.
6. 6 Kluin-Nelemans, H.C., Jansen, J.H., Breukelman, H., et al. Response to interferon alfa-2b in a patient with systemic mastocytosis. N Engl J Med 326 (1992), 619–623.
7. 7 Akin, C., Cladribine for mastocytosis: benefits and risks. Blood 126 (2015), 931–932.
8. 8 Barete, S., Lortholary, O., Damaj, G., et al. Long-term efficacy and safety of cladribine (2-CdA) in adult patients with mastocytosis. Blood 126 (2015), 1009–1016 quiz 1050.
9. 9 Arock, M., Akin, C., Hermine, O., Valent, P., Current treatment options in patients with mastocytosis: status in 2015 and future perspectives. Eur J Haematol 94 (2015), 474–490.
10. 10 Valent, P., Sperr, W.R., Akin, C., How I treat patients with advanced systemic mastocytosis. Blood 116 (2010), 5812–5817.
11. 11 Valent, P., Akin, C., Sperr, W.R., et al. Aggressive systemic mastocytosis and related mast cell disorders: current treatment options and proposed response criteria. Leuk Res 27 (2003), 635–641.
12. 12 Ustun, C., DeRemer, D.L., Akin, C., Tyrosine kinase inhibitors in the treatment of systemic mastocytosis. Leuk Res 35 (2011), 1143–1152.
13. 13 Arock, M., Sotlar, K., Akin, C., et al. KIT mutation analysis in mast cell neoplasms: recommendations of the European Competence Network on Mastocytosis. Leukemia 29 (2015), 1223–1232.
14. 14 Verstovsek, S., Tefferi, A., Cortes, J., et al. Phase II study of dasatinib in Philadelphia chromosome-negative acute and chronic myeloid diseases, including systemic mastocytosis. Clin Cancer Res 14 (2008), 3906–3915.
15. 15 Akin, C., Fumo, G., Yavuz, A.S., et al. A novel form of mastocytosis associated with a transmembrane c-kit mutation and response to imatinib. Blood 103 (2004), 3222–3225.
16. 16 Hochhaus, A., Baccarani, M., Giles, F.J., et al. Nilotinib in patients with systemic mastocytosis: analysis of the phase 2, open-label, single-arm nilotinib registration study. J Cancer Res Clin Oncol 141 (2015), 2047–2060.
17. 17 Randall, N., Courville, E.L., Baughn, L., et al. Bosutinib, a Lyn/Btk inhibiting tyrosine kinase inhibitor, is ineffective in advanced systemic mastocytosis. Am J Hematol, 90, 2015, E74.
18. 18 Ustun, C., Corless, C.L., Savage, N., et al. Chemotherapy and dasatinib induce long-term hematologic and molecular remission in systemic mastocytosis with acute myeloid leukemia with KIT D816V. Leuk Res 33 (2009), 735–741.
19. 19 Gotlib, J., Kluin-Nelemans, H.C., George, T.I., et al. Midostaurin (PKC412) demonstrates a high rate of durable responses in patients with advanced systemic mastocytosis: results from the fully accrued global phase 2 CPKC412D2201 trial. Blood, 124, 2014 abstract 177.
20. 20 Nakamura, R., Chakrabarti, S., Akin, C., et al. A pilot study of nonmyeloablative allogeneic hematopoietic stem cell transplant for advanced systemic mastocytosis. Bone Marrow Transplant 37 (2006), 353–358.
21. 21 Chen, T.Y., Chen, J.S., Huang, W.T., et al. Rapid engraftment of mast cells of donor origin in a case of acute myeloid leukemia with mast cell leukemia after allogeneic stem cell transplantation. Bone Marrow Transplant 32 (2003), 111–114.
22. 22 Gromke, T., Elmaagacli, A.H., Ditschkowski, M., et al. Delayed graft-versus-mast-cell effect on systemic mastocytosis with associated clonal haematological non-mast cell lineage disease after allogeneic transplantation. Bone Marrow Transplant 48 (2013), 732–733.
23. 23 Spyridonidis, A., Thomas, A.K., Bertz, H., et al. Evidence for a graft-versus-mast-cell effect after allogeneic bone marrow transplantation. Bone Marrow Transplant 34 (2004), 515–519.
24. 24 Pullarkat, V., Bedell, V., Kim, Y., et al. Neoplastic mast cells in systemic mastocytosis associated with t(8;21) acute myeloid leukemia are derived from the leukemic clone. Leuk Res 31 (2007), 261–265.
25. 25 Przepiorka, D., Giralt, S., Khouri, I., et al. Allogeneic marrow transplantation for myeloproliferative disorders other than chronic myelogenous leukemia: review of forty cases. Am J Hematol 57 (1998), 24–28.
26. 26 Fodinger, M., Fritsch, G., Winkler, K., et al. Origin of human mast cells: development from transplanted hematopoietic stem cells after allogeneic bone marrow transplantation. Blood 84 (1994), 2954–2959.
27. 27 Ustun, C., Reiter, A., Scott, B.L., et al. Hematopoietic stem-cell transplantation for advanced systemic mastocytosis. J Clin Oncol 32 (2014), 3264–3274.
28. 28 Georgin-Lavialle, S., Lhermitte, L., Dubreuil, P., et al. Mast cell leukemia. Blood 121 (2013), 1285–1295.
29. 29 Majhail, N.S., Farnia, S.H., Carpenter, P.A., et al. Indications for autologous and allogeneic hematopoietic cell transplantation: guidelines from the American Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant 21 (2015), 1863–1869.
30. 30 Nagai, S., Ichikawa, M., Takahashi, T., et al. The origin of neoplastic mast cells in systemic mastocytosis with AML1/ETO-positive acute myeloid leukemia. Exp Hematol 35 (2007), 1747–1752.
31. 31 Ustun, C., Marcucci, G., Emerging diagnostic and therapeutic approaches in core binding factor acute myeloid leukaemia. Curr Opin Hematol 22 (2015), 85–91.
32. 32 Fritsche-Polanz, R., Fritz, M., Huber, A., et al. High frequency of concomitant mastocytosis in patients with acute myeloid leukemia exhibiting the transforming KIT mutation D816V. Mol Oncol 4 (2010), 335–346.
33. 33 Valent, P., Sotlar, K., Sperr, W.R., et al. Refined diagnostic criteria and classification of mast cell leukemia (MCL) and myelomastocytic leukemia (MML): a consensus proposal. Ann Oncol 25 (2014), 1691–1700.
34. 34 Valent, P., Sotlar, K., Sperr, W.R., et al. Chronic mast cell leukemia: a novel leukemia-variant with distinct morphological and clinical features. Leuk Res 39 (2015), 1–5.
35. 35 Horny, H.P., Sotlar, K., Valent, P., Mastocytosis: state of the art. Pathobiology 74 (2007), 121–132.
36. 36 Sotlar, K., Colak, S., Bache, A., et al. Variable presence of KITD816V in clonal haematological non-mast cell lineage diseases associated with systemic mastocytosis (SM-AHNMD). J Pathol 220 (2010), 586–595.
37. 37 Valent, P., Akin, C., Escribano, L., et al. Standards and standardization in mastocytosis: consensus statements on diagnostics, treatment recommendations and response criteria. Eur J Clin Invest 37 (2007), 435–453.
38. 38 Valent, P., Escribano, L., Broesby-Olsen, S., et al. Proposed diagnostic algorithm for patients with suspected mastocytosis: a proposal of the European Competence Network on Mastocytosis. Allergy 69 (2014), 1267–1274.
39. 39 Gotlib, J., Pardanani, A., Akin, C., et al. International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) & European Competence Network on Mastocytosis (ECNM) consensus response criteria in advanced systemic mastocytosis. Blood 121 (2013), 2393–2401.
40. 40 Sperr, W.R., Drach, J., Hauswirth, A.W., et al. Myelomastocytic leukemia: evidence for the origin of mast cells from the leukemic clone and eradication by allogeneic stem cell transplantation. Clin Cancer Res 11:19 Pt 1 (2005), 6787–6792.
41. 41 Arredondo, A.R., Gotlib, J., Shier, L., et al. Myelomastocytic leukemia versus mast cell leukemia versus systemic mastocytosis associated with acute myeloid leukemia: a diagnostic challenge. Am J Hematol 85 (2010), 600–606.
42. 42 Morton, A.J., Gooley, T., Hansen, J.A., et al. Association between pretransplant interferon-alpha and outcome after unrelated donor marrow transplantation for chronic myelogenous leukemia in chronic phase. Blood 92 (1998), 394–401.
43. 43 Sorror, M.L., Maris, M.B., Storb, R., et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood 106 (2005), 2912–2919.
44. 44 Sorror, M.L., Storb, R.F., Sandmaier, B.M., et al. Comorbidity-age index: a clinical measure of biologic age before allogeneic hematopoietic cell transplantation. J Clin Oncol 32 (2014), 3249–3256.
45. 45 Anasetti, C., Logan, B.R., Lee, S.J., et al. Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med 367 (2012), 1487–1496.
46. 46 Holtick, U., Albrecht, M., Chemnitz, J.M., et al. Bone marrow versus peripheral blood allogeneic haematopoietic stem cell transplantation for haematological malignancies in adults. Cochrane Database Syst Rev, 4, 2014, CD010189.
47. 47 Weisdorf, D.J., Nelson, G., Lee, S.J., et al. Sibling versus unrelated donor allogeneic hematopoietic cell transplantation for chronic myelogenous leukemia: refined HLA matching reveals more graft-versus-host disease but not less relapse. Biol Blood Marrow Transplant 15 (2009), 1475–1478.
48. 48 Jagasia, M., Arora, M., Flowers, M.E., et al. Risk factors for acute GVHD and survival after hematopoietic cell transplantation. Blood 119 (2012), 296–307.
49. 49 Gabillot-Carre, M., Lepelletier, Y., Humbert, M., et al. Rapamycin inhibits growth and survival of D816V-mutated c-kit mast cells. Blood 108 (2006), 1065–1072.
50. 50 Cutler, C., Stevenson, K., Kim, H.T., et al. Sirolimus is associated with veno-occlusive disease of the liver after myeloablative allogeneic stem cell transplantation. Blood 112 (2008), 4425–4431.
51. 51 Wimazal, F., Geissler, P., Shnawa, P., et al. Severe life-threatening or disabling anaphylaxis in patients with systemic mastocytosis: a single-center experience. Int Arch Allergy Immunol 157 (2012), 399–405.
52. 52 Akin, C., Anaphylaxis and mast cell disease: what is the risk?. Curr Allergy Asthma Rep 10 (2010), 34–38.
53. 53 Krauth, M.T., Mirkina, I., Herrmann, H., et al. Midostaurin (PKC412) inhibits immunoglobulin E-dependent activation and mediator release in human blood basophils and mast cells. Clin Exp Allergy 39 (2009), 1711–1720.
54. 54 Bejanyan, N., Oran, B., Shanley, R., et al. Clinical outcomes of AML patients relapsing after matched-related donor and umbilical cord blood transplantation. Bone Marrow Transplant 49 (2014), 1029–1035.
55. 55 Rossini, M., Zanotti, R., Viapiana, O., et al. Bone involvement and osteoporosis in mastocytosis. Immunol Allergy Clin North Am 34 (2014), 383–396.
56. 56 Sotlar, K., Bache, A., Stellmacher, F., et al. Systemic mastocytosis associated with chronic idiopathic myelofibrosis: a distinct subtype of systemic mastocytosis associated with a [corrected] clonal hematological non-mast [corrected] cell lineage disorder carrying the activating point mutations KITD816V and JAK2V617F. J Mol Diagn 10 (2008), 58–66.
57. 57 Bastart, F., Escape, I., Solduga, C., et al. Liver infiltration in systemic mastocytosis: ultrasonographic findings. J Ultrasound Med 16 (1997), 223–226.
58. 58 Rossini, M., Zanotti, R., Viapiana, O., et al. Zoledronic acid in osteoporosis secondary to mastocytosis. Am J Med 127 (2014), 1127.e1121–1127.e1124.
59. 59 Alpay Kanitez, N., Erer, B., Dogan, O., et al. Osteoporosis and osteopathy markers in patients with mastocytosis. Turk J Haematol 32 (2015), 43–50.
60. 60 Ustun, C., Smith, A., Cayci, Z., et al. Allogeneic hematopoietic cell transplantation in systemic mastocytosis: is there a high risk for veno-occlusive disease. Eur J Haematol 96 (2016), 655–657.
61. 61 Ghandur-Mnaymneh, L., Gould, E., Systemic mastocytosis with portal hypertension. Autopsy findings and ultrastructural study of the liver. Arch Pathol Lab Med 109 (1985), 76–78.
62. 62 Metcalfe, D.D., The liver, spleen, and lymph nodes in mastocytosis. J Invest Dermatol 96:3 Suppl (1991), 45S–46S discussion 46S, 60S-65S.
63. 63 Fortoul, T.I., Barrios, R., Mast cells and idiopathic lung fibrosis. Arch Invest Med (Mex) 21 (1990), 5–10.
64. 64 Mican, J.M., Di Bisceglie, A.M., Fong, T.L., et al. Hepatic involvement in mastocytosis: clinicopathologic correlations in 41 cases. Hepatology 22:4 Pt 1 (1995), 1163–1170.
65. 65 Grigg, E., Ustun, C., Lee, J., Schade, R., Hepatic manifestations of systemic mastocytosis (SM). Am J Gastroenterol, 105, 2010, S294.
66. 66 Adolf, S., Millonig, G., Seitz, H.K., et al. Systemic mastocytosis: a rare case of increased liver stiffness. Case Reports Hepatol, 2012, 2012, 728172.
67. 67 Kalin, T.V., Meliton, L., Meliton, A.Y., et al. Pulmonary mastocytosis and enhanced lung inflammation in mice heterozygous null for the Foxf1 gene. Am J Respir Cell Mol Biol 39 (2008), 390–399.
68. 68 Carreras, E., How I manage sinusoidal obstruction syndrome after haematopoietic cell transplantation. Br J Haematol 168 (2015), 481–491.
69. 69 Ustun, C., Courville, E.L., Resolution of osteosclerosis after alloHCT in systemic mastocytosis. Blood, 127, 2016, 1836.
70. 70 Stone, R., Mandrekar, S., Sanford, M., et al. The multi-kinase inhibitor midostaurin (M) prolongs survival compared with placebo (P) in combination with daunorubicin (D)/cytarabine (C) induction (ind), high-dose C consolidation (consol), and as maintenance (maint) therapy in newly diagnosed acute myeloid leukemia (AML) patients (pts) age 18-60 with FLT3 mutations (muts): an international prospective randomized (rand) P-controlled double-blind trial (CALGB 10603/RATIFY [Alliance]. [ASH Abstract] Blood, 126, 2015 abstract 6.
71. 71 Dutreix, C., Munarini, F., Lorenzo, S., et al. Investigation into CYP3A4-mediated drug-drug interactions on midostaurin in healthy volunteers. Cancer Chemother Pharmacol 72 (2013), 1223–1234.
72. 72 Akin, C., Soto, D., Brittain, E., et al. Tryptase haplotype in mastocytosis: relationship to disease variant and diagnostic utility of total tryptase levels. Clin Immunol 123 (2007), 268–271.
73. 73 Sperr, W.R., Jordan, J.H., Fiegl, M., et al. Serum tryptase levels in patients with mastocytosis: correlation with mast cell burden and implication for defining the category of disease. Int Arch Allergy Immunol 128 (2002), 136–141.
74. 74 Matito, A., Morgado, J.M., Alvarez-Twose, I., et al. Serum tryptase monitoring in indolent systemic mastocytosis: association with disease features and patient outcome. PLoS One, 8, 2013, e76116.
75. 75 Sperr, W.R., El-Samahi, A., Kundi, M., et al. Elevated tryptase levels selectively cluster in myeloid neoplasms: a novel diagnostic approach and screen marker in clinical haematology. Eur J Clin Invest 39 (2009), 914–923.
76. 76 Erben, P., Schwaab, J., Metzgeroth, G., et al. The KIT D816V expressed allele burden for diagnosis and disease monitoring of systemic mastocytosis. Ann Hematol 93 (2014), 81–88.
77. 77 Hoermann, G., Gleixner, K.V., Dinu, G.E., et al. The KIT D816V allele burden predicts survival in patients with mastocytosis and correlates with the WHO type of the disease. Allergy 69 (2014), 810–813.
78. 78 Schmidt, M., Dercken, C., Loke, O., et al. Pulmonary manifestation of systemic mast cell disease. Eur Respir J 15 (2000), 623–625.
79. 79 Huang, T.Y., Yam, L.T., Li, C.Y., Radiological features of systemic mast-cell disease. Br J Radiol 60 (1987), 765–770.
80. 80 Schwaab, J., Schnittger, S., Sotlar, K., et al. Comprehensive mutational profiling in advanced systemic mastocytosis. Blood 122 (2013), 2460–2466.
81. 81 Damaj, G., Joris, M., Chandesris, O., et al. ASXL1 but not TET2 mutations adversely impact overall survival of patients suffering systemic mastocytosis with associated clonal hematologic non-mast-cell diseases. PLoS One, 9, 2014, e85362.
82. 82 Hanssens, K., Brenet, F., Agopian, J., et al. SRSF2-p95 hotspot mutation is highly associated with advanced forms of mastocytosis and mutations in epigenetic regulator genes. Haematologica 99 (2014), 830–835.
83. 83 Jawhar, M., Schwaab, J., Schnittger, S., et al. Additional mutations in SRSF2, ASXL1 and/or RUNX1 identify a high-risk group of patients with KIT D816V(+) advanced systemic mastocytosis. Leukemia 30 (2016), 136–143.