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

• Published in: Haematologica (Roma) Vol. 109; no. 8; pp. 2619 - 2627
• Main Authors: Garderet, Laurent, Gras, Luuk, Koster, Linda, De Wreede, Liesbeth, Montserrat, Rovira, Vincent, Laure, Fenk, Roland, Karunanithi, Kamaraj, Deeren, Dries, Kaufmann, Martin, Kuball, Jürgen, Ozdogu, Hakan, Cascon, Maria Jesus Pascual, Passweg, Jakob, Rye, Adam, Salmenniemi, Urpu, Snowden, John, Hansen, Charlotte Toftmann, Leleu, Xavier, Gastaud, Lauris, Sokolowska, Joanna Drozd, Raj, Kavita, Beksac, Meral, Schönland, Stefan, Hayden, Patrick, McLornan, Donal
• Format: Journal Article
• Language: English
• Published: Italy Fondazione Ferrata Storti 01.08.2024; Ferrata Storti Foundation
• Subjects: Adult; Aged; Europe; Female; Follow-Up Studies; Glomerular Filtration Rate; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic Stem Cell Transplantation - methods; Humans; Immunoglobulin Light Chains - blood; Male; Middle Aged; Paraproteinemias - diagnosis; Paraproteinemias - mortality; Paraproteinemias - therapy; Plasma Cell Disorders; Registries; Retrospective Studies; Transplantation Conditioning - methods; Transplantation, Autologous; Treatment Outcome; Europe
• ISSN: 0390-6078; 1592-8721; 1592-8721
• DOI: 10.3324/haematol.2023.284520

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 European Society for Blood and Bone Marrow transplantation 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 100 mg/m2 in 23%, 140 mg/m2 in 55% and 200 mg/m2 in 21%. The rate of very good partial response or better improved from 41% pretransplant 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 estimated glomerular filtration rate 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 (interquartile range [IQR]: 46-122). At 6-years post ASCT, overall survival was 88% (95% confidence interval [CI]: 78-98) and PFS was 44% (95% CI: 28-60). The 2-year cumulative incidence of relapse and non-relapse mortality 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 favorable with low non-relapse mortality and good long-term overall survival.