PMID- 38546696
OWN - NLM
STAT- Publisher
LR  - 20240328
IS  - 1592-8721 (Electronic)
IS  - 0390-6078 (Linking)
DP  - 2024 Mar 28
TI  - 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.
LID - 10.3324/haematol.2023.284520 [doi]
AB  - 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 mumol/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.
FAU - Garderet, Laurent
AU  - Garderet L
AD  - Sorbonne University, APHP, Hopital Pitie Salpetriere, service d'hematologie, 
      Paris. laurent.garderet@aphp.fr.
FAU - Gras, Luuk
AU  - Gras L
AD  - EBMT Statistical Unit, Leiden.
FAU - Koster, Linda
AU  - Koster L
AD  - EBMT Leiden Study Unit, Leiden.
FAU - De Wreede, Liesbeth
AU  - De Wreede L
AD  - Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden.
FAU - Montserrat, Rovira
AU  - Montserrat R
AD  - Hospital clinic, Barcelona.
FAU - Vincent, Laure
AU  - Vincent L
AD  - Clinical Hematology, Montpellier University Hospital Center, Montpellier.
FAU - Fenk, Roland
AU  - Fenk R
AD  - Department of Hematology, Oncology and Clinical Immunology, University Hospital 
      Duesseldorf, Dusseldorf.
FAU - Karunanithi, Kamaraj
AU  - Karunanithi K
AD  - University hospital of North Staffordshire, Stoke.
FAU - Deeren, Dries
AU  - Deeren D
AD  - AZ Delta, Roeselare.
FAU - Kaufmann, Martin
AU  - Kaufmann M
AD  - Robert Bosch Krankenhaus, Stuttgart.
FAU - Kuball, Jurgen
AU  - Kuball J
AD  - Department of Hematology, University Medical Center Utrecht, Utrecht.
FAU - Ozdogu, Hakan
AU  - Ozdogu H
AD  - Department of Hematology, Baskent University Hospital, Adana.
FAU - Cascon, Maria Jesus Pascual
AU  - Cascon MJP
AD  - Hospital Regional de Malaga, Malaga.
FAU - Passweg, Jakob
AU  - Passweg J
AD  - University Hospital Basel, Basel.
FAU - Rye, Adam
AU  - Rye A
AD  - Gloucestershire Hospitals NHS Foundation Trust, Cheltenham.
FAU - Salmenniemi, Urpu
AU  - Salmenniemi U
AD  - HUCH Comprehensive Cancer Center, Stem Cell Transplantation Unit - Helsinki.
FAU - Snowden, John
AU  - Snowden J
AD  - Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield.
FAU - Hansen, Charlotte Toftmann
AU  - Hansen CT
AD  - Department of Hematology, Odense University Hospital, Odense.
FAU - Leleu, Xavier
AU  - Leleu X
AD  - CHU Poitiers, Poitiers.
FAU - Gastaud, Lauris
AU  - Gastaud L
AD  - Centre Antoine Lacassagne, Tourrettes-sur-Loup.
FAU - Sokolowska, Joanna Drozd
AU  - Sokolowska JD
AD  - Central Clinical Hospital, The Medical University of Warsaw, Warsaw, Poland.
FAU - Raj, Kavita
AU  - Raj K
AD  - University College London Hospitals NHS Trust, London.
FAU - Beksac, Meral
AU  - Beksac M
AD  - Ankara University School of Medicine, Hematology Department, Ankara.
FAU - Schonland, Stefan
AU  - Schonland S
AD  - University Hospital Heidelberg, Heidelberg.
FAU - Hayden, Patrick
AU  - Hayden P
AD  - Department of Haematology, Trinity College Dublin, St. James's Hospital, Dublin.
FAU - McLornan, Donal
AU  - McLornan D
AD  - University College London Hospitals NHS Trust, London.
LA  - eng
PT  - Journal Article
DEP - 20240328
PL  - Italy
TA  - Haematologica
JT  - Haematologica
JID - 0417435
SB  - IM
EDAT- 2024/03/28 12:50
MHDA- 2024/03/28 12:50
CRDT- 2024/03/28 11:44
PHST- 2023/10/22 00:00 [received]
PHST- 2024/03/28 12:50 [medline]
PHST- 2024/03/28 12:50 [pubmed]
PHST- 2024/03/28 11:44 [entrez]
AID - 10.3324/haematol.2023.284520 [doi]
PST - aheadofprint
SO  - Haematologica. 2024 Mar 28. doi: 10.3324/haematol.2023.284520.