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Blood, 15 March 2006, Vol. 107, No. 6, pp. 2578-2584.
Prepublished online as a Blood First Edition Paper on November 17, 2005; DOI 10.1182/blood-2005-06-2462.
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Submitted June 21, 2005
Accepted November 2, 2005
Allogeneic and syngeneic hematopoietic cell transplantation in patients with amyloid light chain amyloidosis: a report from the European Group for Blood and Marrow Transplantation
Stefan O Schonland*, Henk Lokhorst, Agnes Buzyn, Veronique Leblond, Ute Hegenbart, Giuseppe Bandini, Andrew Campbell, Enric Carreras, Augustin Ferrant, Leanthe Grommisch, Peter Jacobs, Nicolaus Kroger, Giorgio La Nasa, Nigel Russell, Pierre Zachee, Hartmut Goldschmidt, Simona Iacobelli, Dietger Niederwieser, and Gosta Gahrton
University of Heidelberg, Heidelberg, Germany
University Medical Centre Utrecht, Utrecht, The Netherlands
Hopital Necker Paris, Paris, France
Pitie Salpetriere, Paris, France
Institute of Hematology, Bologna University, Bologna, Italy
Oxford Radcliffe Hospital, Oxford, United Kingdom
Institute of Hematology, Barcelona, Spain
Cliniques Universitaires St. Luc, Brussels, Belgium
University of Leipzig, Leipzig, Germany
Department of Hematology and BMT Unit, Constantiaberg Medi-Clinic, Cape Town, South Africa
BMT Centre, University Hospital Eppendorf, Hamburg, Germany
Centro Trapianti di Midollo Osseo, Cagliari, Italy
Nottingham City Hospital, Nottingham, United Kingdom
AZ Stuivenberg, Antwerpen, Belgium
Dept. of Medical Statistics, European Research Support Office, Leiden, The Netherlands
Karolinska University Hospital, Stockholm, Sweden
* Corresponding author; email: stefan_schoenland{at}med.uni-heidelberg.de.
Using the EBMT registry, we retrospectively studied 19 patients with AL (amyloid light chain) amyloidosis who underwent allogeneic (allo-; n=15) or syngeneic (syn-; n=4) hematopoietic stem cell transplantation (SCT) between 1991 and 2003. For allo-SCT, full-intensity conditioning was used in 7 patients and reduced-intensity conditioning (RIC) in 8 patients. Engraftment was durable in 12 of those 15 patients. The median follow-up time is 19 months. Kaplan-Meier probabilities of overall and progression-free survival were 60 and 53% at 1 year, respectively. Overall, 40% of patients died of treatment-related mortality (TRM). Best hematological response after SCT was complete remission (CR) and partial remission (PR) in 8 and 2 patients, respectively, leading to an organ response in 8 of these patients. Seven of the 10 patients in remission are long-term survivors. In 5 out of 7 evaluable patients with CR chronic graft-versus-host disease (GvHD) was observed indicating the contribution of immune effects to disease control. Main clinical problem was cardiac failure in patients with poor performance status due to amyloidosis or in combination with severe infections. These data suggest that allo-SCT might be a promising and potentially curative treatment modality for selected patients with AL amyloidosis.
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