Autologous stem cell transplantation in Waldenström macroglobulinemia
Authors:
M. Kaščák 1; J. Ďuraš 1; M. Navrátil 1; R. Hájek 1,2,3
Authors‘ workplace:
Klinika hematoonkologie, Fakultní nemocnice Ostrava
1; Lékařská fakulta, Ostravská univerzita, Ostrava
2; Babákova myelomová skupina, Ústav patologické fyziologie, Masarykova univerzita, Brno
3
Published in:
Transfuze Hematol. dnes,22, 2016, No. 1, p. 28-38.
Category:
Comprehensive Reports, Original Papers, Case Reports
Overview
Waldenström macroglobulinemia is a rare incurable monoclonal B lymphoproliferative disease, characterized by bone marrow infiltration and production of IgM paraprotein. Given its clinical course, it is indolent in nature, with a median overall survival of 6 years. Primary treatment using rituximab based regimens has overall response rates of 80% to 90% with median remission duration of 3 to 4 years. High dose chemotherapy with rescue autologous stem cell transplantation is an effective modality for treating patients with Waldenström macroglobulinemia. However, the issue of correct timing and selection of a suitable candidate for autologous transplantation remains unclear. There is no consensus regarding the most appropriate conditioning myeloablative regimen. High dose melphalan, carmustine or thiotepa based chemotherapy regimens and total-body irradiation based regimens are most commonly used. Autologous stem cell transplantation is a safe procedure with a low 1 year post-transplant mortality, usually not exceeding 5% even in an elderly patient population. In relapsed Waldenström macroglobulinemia, autologous transplantation has overall response rates of 80% to 90% with 20% f complete remissions. The expected median duration of remission is 3 to 4 years with median overall survival exceeding 60% after 5 years. Autologous transplantation should always be considered for the treatment of younger patients in first chemosensitive relapse and with a short duration of remission (less than 1–2 years) as well as in patients with a high risk prognostic score. In the future, it is expected that autologous transplantation will be used to treat early stage disease, at the time of best achieved response, similar to multiple myeloma treatment. This paper summarizes current knowledge regarding the use of autologous stem cell transplantation in Waldenström macroglobulinemia.
Key words:
Waldenström macroglobulinemia – autologous stem cell transplantation – high dose therapy
Sources
1. Owen RG, Treon SP, Al-Katib A, et al. Clinicopathological definition of Waldenstrom’s macroglobulinemia: consensus panel recommenda-tions from the Second International Workshop on Waldenstrom’s Macroglobulinemia. Semin Oncol 2003; 30: 110–115.
2. Swerdlow S. WHO Classification of Tumours of Haematopoietic and Lymphoid Tissues, 4th ed. International Agency for Research on Cancer: Lyon, France, 2008, p. 194.
3. Groves FD, Travis LB, Devesa SS, et al. Waldenström’s macroglobulinemia: incidence patterns in the United States, 1988–1994. Cancer 1998; 82: 1078–1081.
4. Phekoo KJ, Jack RH, Davies E, et al. The incidence and survival of Waldenström’s macroglobulinaemia in South East England. Leuk Res 2008; 32: 55–59.
5. Ghobrial IM, Fonseca R, Gertz MA, et al. Prognostic model for disease-specific and overall mortality in newly diagnosed symptomatic patients with Waldenstrom macroglobulinaemia. Br J Haematol 2006; 133: 158–164.
6. Wang H, Chen Y, Li F, et al. Temporal and geographic variations of Waldenstrom macroglobulinemia incidence: a large population-based study. Cancer 2012; 118: 3793–3800.
7. Treon SP. How I treat Waldenström macroglobulinemia. Blood 2009; 114: 2375–2385.
8. Dimopoulos MA, Kyle RA, Anagnostopoulos A, et al. Diagnosis and management of Waldenstrom’s macroglobulinemia. J Clin Oncol 2005; 23: 1564–1577.
9. Kyle RA, Benson JT, Larson DR, et al. Progression in smoldering Waldenstrom macroglobulinemia: long-term results. Blood 2012; 119: 4462–4466.
10. Ansell SM, Kyle RA, Reeder CB, et al. Diagnosis and management of Waldenström macroglobulinemia: Mayo stratification of macroglobulinemia and risk-adapted therapy (mSMART) guidelines. Mayo Clin Proc 2010; 85: 824–833.
11. Owen RG, Pratt G, Auer RL, et al. Guidelines on the diagnosis and management of Waldenström macroglobulinaemia. Br J Haematol 2014; 165: 316–333.
12. Yang L, Wen B, Li H, et al. Autologous peripheral blood stem cell transplantation for Waldenstrom’s macroglobulinemia. Bone Marrow Transplant 1999; 24: 929–930.
13. Treon SP. How I treat Waldenström macroglobulinemia. Blood 2015; 126: 721–732.
14. Brito-Babapulle F, Bowcock SJ, Marcus RE, et al. Autografting for patients with chronic myeloid leukaemia in chronic phase: peripheral blood stem cells may have a finite capacity for maintaining haemopoiesis. Br J Haematol 1989; 73: 76–81.
15. Desikan R, Dhodapkar M, Siegel D, et al. High-dose therapy with autologous haemopoietic stem cell support for Waldenström’s macroglobulinaemia. Br J Haematol 1999; 105: 993–996.
16. Anagnostopoulos A, Dimopoulos MA, Aleman A, et al. High-dose chemotherapy followed by stem cell transplantation in patients with resistant Waldenstrom’s macroglobulinemia. Bone Marrow Transplant 2001; 27: 1027–1029.
17. Munshi NC, Barlogie B. Role for high-dose therapy with autologous hematopoietic stem cell support in Waldenstrom’s macroglobulinemia. Semin Oncol 2003; 30: 282–285.
18. Fassas A, Barlogie B, Ward S, et al. High-dose treatment (HDT) and autologous stem cell transplant (ASCT) in Waldenstrom’s macroglobulinemia (WM) patients (pts): A single center experience. ASCO Meet Abstr 2005; 23: 6661.
19. Akhtar S, Weshi AE, Rahal M, et al. Factors affecting autologous peripheral blood stem cell collection in patients with relapsed or refractory diffuse large cell lymphoma and Hodgkin lymphoma: a single institution result of 168 patients. Leuk Lymphoma 2008; 49: 769–778.
20. Perea G, Sureda A, Martino R, et al. Predictive factors for a successful mobilization of peripheral blood CD34+ cells in multiple myeloma. Ann Hematol 2001; 80: 592–597.
21. Gratwohl A, Baldomero H, Schmid O, et al. Change in stem cell source for hematopoietic stem cell transplantation (HSCT) in Europe: a report of the EBMT activity survey 2003. Bone Marrow Transplant 2005; 36: 575–590.
22. Sheppard D, Bredeson C, Allan D, et al. Systematic review of randomized controlled trials of hematopoietic stem cell mobilization strategies for autologous transplantation for hematologic malignancies. Biol Blood Marrow Transplant 2012; 18: 1191–1203.
23. Watanabe H, Watanabe T, Suzuya H, et al. Peripheral blood stem cell mobilization by granulocyte colony-stimulating factor alone and engraftment kinetics following autologous transplantation in children and adolescents with solid tumor. Bone Marrow Transplant 2006; 37: 661–668.
24. Gojo I, Guo C, Sarkodee-Adoo C, et al. High-dose cyclophosphamide with or without etoposide for mobilization of peripheral blood progenitor cells in patients with multiple myeloma: efficacy and toxicity. Bone Marrow Transplant 2004; 34: 69–76.
25. Kyriakou C, Canals C, Sibon D, et al. High-dose therapy and autologous stem-cell transplantation in Waldenstrom macroglobulinemia: the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol 2010; 28: 2227–2232.
26. Caravita T, Siniscalchi A, Tendas A, et al. High-dose therapy with autologous PBSC transplantation in the front-line treatment of Waldenstrom’s macroglobulinemia. Bone Marrow Transplant 2009; 43: 587–588.
27. Gertz MA, Reeder CB, Kyle RA, et al. Stem cell transplant for Waldenström macroglobulinemia: an underutilized technique. Bone Marrow Transplant 2012; 47: 1147–1153.
28. Flomenberg N, Devine SM, Dipersio JF, et al. The use of AMD3100 plus G-CSF for autologous hematopoietic progenitor cell mobiliza-tion is superior to G-CSF alone. Blood 2005; 106: 1867–1874.
29. Kyriakou C, Canals C, Taghipour G, et al. Autologous stem cell transplantation (ASCT) for patients with Waldenstrom’s macroglobulinaemia: an analysis of 201 cases from the European Bone Marrow Transplant Registry (EBMT) [abstract]. Haematologica 2007; 92 (S2): 228.
30. Shaw PJ, Nath CE, Lazarus HM. Not too little, not too much-just right! (Better ways to give high dose melphalan). Bone Marrow Transplant 2014; 49: 1457–1465.
31. Palumbo A, Bringhen S, Bruno B, et al. Melphalan 200 mg/m2 versus melphalan 100 mg/m2 in newly diagnosed myeloma patients: a prospective, multicenter phase 3 study. Blood 2010; 115: 1873–1879.
32. Raab MS, Breitkreutz I, Hundemer M, et al. The outcome of autologous stem cell transplantation in patients with plasma cell disorders and dialysis-dependent renal failure. Haematologica 2006; 91: 1555–1558.
33. Caballero MD, Rubio V, Rifon J, et al. BEAM chemotherapy followed by autologous stem cell support in lymphoma patients: analysis of efficacy, toxicity and prognostic factors. Bone Marrow Transplant 1997; 20: 451–458.
34. Dreger P, Glass B, Kuse R, et al. Myeloablative radiochemotherapy followed by reinfusion of purged autologous stem cells for Waldenström’s macroglobulinaemia. Br J Haematol 1999; 106: 115–118.
35. Tournilhac O, Leblond V, Tabrizi R, et al. Transplantation in Waldenstrom’s macroglobulinemia – the French experience. Semin Oncol 2003; 30: 291–296.
36. Anagnostopoulos A, Hari PN, Pérez WS, et al. Autologous or allogeneic stem cell transplantation in patients with Waldenstrom’s macroglobulinemia. Biol Blood Marrow Transplant 2006; 12: 845–854.
37. Gilleece MH, Pearce R, Linch DC, et al. The outcome of haemopoietic stem cell transplantation in the treatment of lymphoplasmacytic lymphoma in the UK: a British Society Bone Marrow Transplantation study. Hematol Amst Neth 2008; 13: 119–127.
38. Anagnostopoulos A, Giralt S. Stem cell transplantation (SCT) for Waldenstrom’s macroglobulinemia (WM). Bone Marrow Transplant 2002; 29: 943–947.
39. Dhedin N, Tabrizi R, Bulabois PE, Le Gouill S, Coiteux V, Dartigeas C et al. Hematopoietic stem cell transplantation (HSCT) in Waldenstrom macroglobulinemia (WM): update of the French experience in 54 cases. Haematologica 2007; 92 (S): 228.
40. Dreger P, Schmitz N. Autologous stem cell transplantation as part of first-line treatment of Waldenström’s macroglobulinemia. Biol Blood Marrow Transplant 2007; 13: 623–624.
41. Kyriakou C. Optimal timing for stem cell transplantation (SCT) in Waldenstrom macroglobulinemia. International Workshop for Waldenstrom Macroglobulinemia, London 2014. Dostupné na www: http://www.wmworkshop.org/conferences/london-2014/170-conferences/london-2014/822.
42. Treon SP, Ioakimidis L, Soumerai JD, et al. Primary therapy of Waldenström macroglobulinemia with bortezomib, dexamethasone, and rituximab: WMCTG clinical trial 05-180. J Clin Oncol 2009; 27: 3830–3835.
43. Dimopoulos MA, Anagnostopoulos A, Kyrtsonis MC, et al. Primary treatment of Waldenström macroglobulinemia with dexamethasone, rituximab, and cyclophosphamide. J Clin Oncol 2007; 25: 3344–3349.
44. Ioakimidis L, Patterson CJ, Hunter ZR, et al. Comparative outcomes following CP-R, CVP-R, and CHOP-R in Waldenström’s macroglobulinemia. Clin Lymphoma Myeloma 2009; 9: 62–66.
45. Treon SP, Tripsas CK, Meid K, et al. Carfilzomib, rituximab, and dexamethasone (CaRD) treatment offers a neuropathy-sparing approach for treating Waldenström’s macroglobulinemia. Blood 2014; 124: 503–510.
46. Treon SP, Soumerai JD, Branagan AR, et al. Thalidomide and rituximab in Waldenstrom macroglobulinemia. Blood 2008; 112: 4452–4457.
47. Dimopoulos MA, Roussou M, Kastritis E, et al. Primary treatment of Waldenstrom’s macroglobulinemia with dexamethasone, rituximab and cyclophosphamide (DRC): Final analysis of a phase II study. Blood 2012; 120(21): Abstract 438.
48. Rummel MJ, Niederle N, Maschmeyer G, et al. Bendamustine plus rituximab versus CHOP plus rituximab as first-line treatment for patients with indolent and mantle-cell lymphomas: an open-label, multicentre, randomised, phase 3 non-inferiority trial. Lancet 2013; 381: 1203–1210.
49. Dimopoulos MA, Kastritis E, Owen RG, et al. Treatment recommendations for patients with Waldenström macroglobulinemia (WM) and related disorders: IWWM-7 consensus. Blood 2014; 124: 1404–1411.
50. Chen CI, Kouroukis CT, White D, et al. Bortezomib is active in pa-tients with untreated or relapsed Waldenstrom’s macroglobulinemia: a phase II study of the National Cancer Institute of Canada Clinical Trials Group. J Clin Oncol 2007; 25: 1570–1575.
51. Agathocleous A, Rohatiner A, Rule S, et al. Weekly versus twice weekly bortezomib given in conjunction with rituximab, in patients with recurrent follicular lymphoma, mantle cell lymphoma and Waldenström macroglobulinaemia. Br J Haematol 2010; 151: 346–353.
52. Treon SP, Hanzis C, Tripsas C, et al. Bendamustine therapy in pa-tients with relapsed or refractory Waldenström’s macroglobulinemia. Clin Lymphoma Myeloma Leuk 2011; 11: 133–135.
53. Treon SP, Tripsas CK, Meid K, et al. Ibrutinib in previously treated Waldenström’s macroglobulinemia. N Engl J Med 2015; 372: 1430–1440.
54. Morel P, Duhamel A, Gobbi P, et al. International prognostic scoring system for Waldenstrom macroglobulinemia. Blood 2009; 113: 4163–4170.
55. Kastritis E, Kyrtsonis M-C, Hadjiharissi E, et al. Validation of the International Prognostic Scoring System (IPSS) for Waldenstrom’s macroglobulinemia (WM) and the importance of serum lactate dehydrogenase (LDH). Leuk Res 2010; 34: 1340–1343.
56. Bachanova V, Burns LJ. Hematopoietic cell transplantation for Waldenström macroglobulinemia. Bone Marrow Transplant 2012; 47: 330–336.
57. Buske C, Leblond V, Dimopoulos M, et al. Waldenstrom’s macroglobulinaemia: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol 2013; 24 Suppl 6: vi155–159.
58. Adam Z, Hájek R, Krejčí M, et al. Diagnostika a léčba Waldenströmovy makroglobulinemie: souhrn doporučení 2014. Transfuze Hematol dnes 2014; 20(Suppl): 5–22.
Labels
Haematology Internal medicine Clinical oncologyArticle was published in
Transfusion and Haematology Today
2016 Issue 1
Most read in this issue
- Prognostic factors in chronic myeloid leukemia: can we gain more from combination?
- Chronic myeloid leukaemia – standardization of BCR-ABL1 transcript level monitoring in the Czech Republic
- Consensual guidelines of the Czech National Haemophilia Programme (CNHP) for factor replacement therapy in patients with haemophilia during invasive procedures and surgery
- Autologous stem cell transplantation in Waldenström macroglobulinemia