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Allogeneic hematopoietic stem cell transplantation allows long-term complete remission and curability in high-risk Waldenström’s macroglobulinemia. Results of a retrospective analysis of the Société Française de Greffe de Moelle et de Thérapie Cellulaire

¹ Service d’Hématologie et de Thérapie Cellulaire, CHU Clermont-Ferrand, Hôp Hotel Dieu, F-63003 and Univ Clermont 1, EA3846, F-63001, France Clermont-Ferrand, France
² Bone marrow transplant unit, Hospital saint Louis, Paris , France
³ Hematology Department, Besançon, France
⁴ Statistical unit, Hospital de la Pitié-Salpêtrière, AP-HP, Paris, France
⁵ Hematology Department Hospital Hôtel-Dieu, Nantes, France
⁶ Hematology Department, CHU Lille
⁷ Hematology Department , Hospital du Haut Leveque Pessac
⁸ Hematology Department, Hospital of Grenoble, France
⁹ Hematology Department, Hospital Henri Mondor, Créteil
¹⁰ Hematology Department, Hospital of Poitiers, France
¹¹ University of Heidelberg, Heidelberg, Germany
¹² Hematology Department, Hotel Dieu, Paris, France
¹³ Hematology Department, CHU Angers, France
¹⁴ Hematology Department, CHU Strasbourg, France
¹⁵ Hematology Department, Hôpital de la Pitié-Salpêtrière, Paris, France
¹⁶ Bone marrow transplant unit, Institut Paoli Calmettes, Marseille

Correspondence: Dr. Nathalie Dhédin, Service d’Hématologie Clinique, Hôpital Pitié-Salpêtrière, 83 bd de l’Hôpital, 75651 Paris Cedex, France. Phone: international + 33.1.42162794. Fax: international + 33.1.42162787. E-mail: nathalie.dhedin{at}psl.aphp.fr

ABSTRACT

BACKGROUND: Patients with poor-risk Waldenström’s macroglobulinemia (WM) have suboptimal response and early post-treatment relapse with conventional therapies. Hence, new therapeutic approaches such as allogeneic stem cell transplantation (SCT) should be evaluated in these patients.

DESIGN AND METHODS: We examined the long-term outcome of allogeneic SCT in WM by studying the records of 24 patients reported in the SFGM-TC database and one transplanted in the bone marrow unit in Hamburg.

RESULTS: Median age at the time of SCT was 48 years (range, 24–64). The patients had previously received a median of 3 lines of therapy (range, 1–6) and 44% of them had refractory disease at time of transplant. Allogeneic SCT after myeloablative (n=12) or reduced-intensity (n=13) conditioning (RIC) yielded an overall response rate of 92% and immunofixation-negative complete remission in 50% of evaluable patients. With a median follow-up of 64 months among survivors (range, 11–149 months), 5-year overall survival and progression-free survival rates were respectively, 67% (95% CI: 46–81) and 58% (95% CI: 38–75). The 5-year estimated risk of progression was 25% (95% CI: 10–36%), with only one relapse among the 12 patients who entered CR, versus 5 in the 12 patients who did not. Only 1 of the 6 relapses occurred more than 3 years post-transplant.

CONCLUSIONS: Allogeneic SCT therefore yields a high rate of complete remissions and is potentially curative in poor-risk WM.

Key words: allogeneic stem cell transplantation, Waldenström’s macroglobulinemia, complete remission.