PMID- 16862166
OWN - NLM
STAT- MEDLINE
DCOM- 20060925
LR  - 20131121
IS  - 0268-3369 (Print)
IS  - 0268-3369 (Linking)
VI  - 38
IP  - 5
DP  - 2006 Sep
TI  - Clinical and molecular characteristics of patients with non-amyloid light chain 
      deposition disorders, and outcome following treatment with high-dose melphalan 
      and autologous stem cell transplantation.
PG  - 339-43
AB  - Light chain deposition disease (LCDD) is caused by a clonal plasma cell disorder 
      in which fragments of monoclonal immunoglobulin light chains form non-fibrillary 
      deposits in various tissues resulting in organ dysfunction. Crystal storing 
      histiocytosis (CSH) is another light chain deposition disorder in which 
      monoclonal light chains form intracytoplasmic crystals. Both are uncommon 
      diseases for which there is limited treatment experience. Between 2003 and 2005, 
      five patients with LCDD and one with CSH were treated at Boston University 
      Medical Center with high-dose melphalan and autologous peripheral blood stem cell 
      transplantation (HDM/SCT). Five of the six patients had predominantly renal 
      involvement, and one patient with LCDD had biopsy-proven deposits in the 
      myocardium. Molecular characterization revealed that the pathologic light chains 
      were kappa in four of the six patients, and sequence analysis revealed unusual 
      germline donor genes and high rates of amino-acid substitutions. One light chain 
      sequence encoded a new potential N-linked glycosylation site, and another showed 
      evidence of antigen selection. All patients are alive and five of the six 
      patients are in complete hematologic remission at a median follow-up of 12 months 
      (range 4-29 months) after HDM/SCT. In our experience, HDM/SCT is a feasible and 
      effective treatment approach for these disorders.
FAU - Weichman, K
AU  - Weichman K
AD  - Section of Hematology/Oncology, Department of Medicine, Boston University Medical 
      Center, Boston, MA 02118, USA.
FAU - Dember, L M
AU  - Dember LM
FAU - Prokaeva, T
AU  - Prokaeva T
FAU - Wright, D G
AU  - Wright DG
FAU - Quillen, K
AU  - Quillen K
FAU - Rosenzweig, M
AU  - Rosenzweig M
FAU - Skinner, M
AU  - Skinner M
FAU - Seldin, D C
AU  - Seldin DC
FAU - Sanchorawala, V
AU  - Sanchorawala V
LA  - eng
GR  - HL 68705/HL/NHLBI NIH HHS/United States
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
PT  - Research Support, Non-U.S. Gov't
DEP - 20060724
PL  - England
TA  - Bone Marrow Transplant
JT  - Bone marrow transplantation
JID - 8702459
RN  - 0 (Immunoglobulin kappa-Chains)
RN  - 0 (Immunoglobulin lambda-Chains)
RN  - Q41OR9510P (Melphalan)
SB  - IM
MH  - Adult
MH  - Histiocytosis/therapy
MH  - Humans
MH  - Immunoglobulin kappa-Chains/*metabolism
MH  - Immunoglobulin lambda-Chains/*metabolism
MH  - Kidney Diseases/*therapy
MH  - Male
MH  - Melphalan/administration & dosage/*therapeutic use
MH  - Middle Aged
MH  - *Stem Cell Transplantation
MH  - Transplantation, Autologous
MH  - Treatment Outcome
EDAT- 2006/07/25 09:00
MHDA- 2006/09/26 09:00
CRDT- 2006/07/25 09:00
PHST- 2006/07/25 09:00 [pubmed]
PHST- 2006/09/26 09:00 [medline]
PHST- 2006/07/25 09:00 [entrez]
AID - 1705447 [pii]
AID - 10.1038/sj.bmt.1705447 [doi]
PST - ppublish
SO  - Bone Marrow Transplant. 2006 Sep;38(5):339-43. doi: 10.1038/sj.bmt.1705447. Epub 
      2006 Jul 24.