melphalan and Histiocytosis

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.

Topics: Adult; Histiocytosis; Humans; Immunoglobulin kappa-Chains; Immunoglobulin lambda-Chains; Kidney Diseases; Male; Melphalan; Middle Aged; Stem Cell Transplantation; Transplantation, Autologous; Treatment Outcome

Necrobiotic xanthogranuloma (NXG) is a rare non-X histiocytosis with conspicuous lesions on the periorbital skin.. A diabetic patient presented with NXG and a previous diagnosis of necrobiosis lipoidica on the legs over a period of almost 2 years before the development of the typical lesions of NXG on the periorbital regions, back, thighs and legs. The patient was found also to have developed lambda paraproteinemia.. Treatment with melphalan and prednisone resulted in great improvement of cutaneous lesions and paraproteinemia remission.. This case report details how melphalan and prednisone can be administered in the successful treatment of necrobiotic xanthogranuloma with lambda paraproteinemia.

Topics: Adult; Anti-Inflammatory Agents; Diagnosis, Differential; Drug Therapy, Combination; Female; Histiocytosis; Humans; Melphalan; Necrobiosis Lipoidica; Paraproteinemias; Prednisone