Long-term outcomes and renal responses following autologous hematopoietic stem cell transplantation for light chain deposition disease: a retrospective study on behalf of the Chronic Malignancies Working Party of the European Society for Blood and Marrow Transplantation
Laurent Garderet,
Luuk Gras,
Linda Koster,
Liesbeth de Wreede,
Rovira Montserrat,
Laure Vincent,
Roland Fenk,
Kamaraj Karunanithi,
Dries Deeren,
Martin Kaufmann,
Jürgen Kuball,
Hakan Ozdogu,
Maria Jesus Pascual Cascon,
Jakob Passweg,
Adam Rye,
Urpu Salmenniemi,
John Snowden,
Charlotte Toftmann Hansen,
Xavier Leleu,
Lauris Gastaud,
Joanna Drozd Sokolowska,
Kavita Raj,
Meral Beksac,
Stefan Schönland,
Patrick Hayden,
Donal McLornan
Affiliations
Laurent Garderet
Sorbonne University, APHP, Hôpital Pitié Salpêtrière, service d’hématologie, Paris
Luuk Gras
EBMT Statistical Unit, Leiden
Linda Koster
EBMT Leiden Study Unit, Leiden
Liesbeth de Wreede
Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden
Rovira Montserrat
Hospital clinic, Barcelona
Laure Vincent
Clinical Hematology, Montpellier University Hospital Center, Montpellier
Roland Fenk
Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Düsseldorf
Kamaraj Karunanithi
University hospital of North Staffordshire, Stoke
Dries Deeren
AZ Delta, Roeselare
Martin Kaufmann
Robert Bosch Krankenhaus, Stuttgart
Jürgen Kuball
Department of Hematology, University Medical Center Utrecht, Utrecht
Hakan Ozdogu
Department of Hematology, Baskent University Hospital, Adana
Maria Jesus Pascual Cascon
Hospital Regional de Málaga, Malaga
Jakob Passweg
University Hospital Basel, Basel
Adam Rye
Gloucestershire Hospitals NHS Foundation Trust, Cheltenham
Urpu Salmenniemi
HUCH Comprehensive Cancer Center, Stem Cell Transplantation Unit – Helsinki
John Snowden
Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield
Charlotte Toftmann Hansen
Department of Hematology, Odense University Hospital, Odense
Xavier Leleu
CHU Poitiers, Poitiers
Lauris Gastaud
Centre Antoine Lacassagne, Tourrettes-sur-Loup
Joanna Drozd Sokolowska
Central Clinical Hospital, The Medical University of Warsaw, Warsaw, Poland
Kavita Raj
University College London Hospitals NHS Trust, London
Meral Beksac
Ankara University School of Medicine, Hematology Department, Ankara
Stefan Schönland
University Hospital Heidelberg, Heidelberg
Patrick Hayden
Department of Haematology, Trinity College Dublin, St. James’s Hospital, Dublin
Donal McLornan
University College London Hospitals NHS Trust, London
There is little long-term outcome data on the efficacy of autologous hematopoietic stem cell transplantation (ASCT) in light chain deposition disease (LCDD). We identified 51 LCDD patients in the EBMT registry who had undergone upfront ASCT between 1995 and 2021. The median serum creatinine was 280 μmol/L and 45% required renal replacement therapy (RRT) at time of transplant. The melphalan dose was 100mg/m2 in 23%, 140mg/m2 in 55% and 200 mg/m2 in 21%. The rate of very good partial response or better improved from 41% pre-transplant to 66% at Day +100 post-ASCT. In RRT-independent patients, there was a modest improvement in renal function within the first 3 months; the median eGFR increased from 44 to 51 ml/min/1.73 m2. There was no further change between 3 and 12 months post- ASCT. No patient who was RRT-independent at ASCT became RRT dependent by Day + 100 post-ASCT. Median follow-up post-ASCT was 84 months (IQR: 46-122). At 6-years post ASCT, overall survival (OS) was 88% (95% CI: 78-98%) and PFS was 44% (95% CI: 28-60%). The 2-year cumulative incidence of relapse and non-relapse mortality (NRM) was 17% (95% CI: 6-27%) and 2% (95% CI: 0-6%), respectively. The cumulative incidence of renal transplantation at 4 years after ASCT was 27% (95% CI 13-41) with renal transplantation performed between 6.3 and 52.9 months post-ASCT (median 24.7 months). ASCT represents a feasible option for LCDD patients even if RRT dependent at time of transplant. Outcomes are favourable with low NRM and good long-term OS.