title: Use of bortezomib in heavy-chain deposition disease: a report of 3 cases.
abstract: Heavy-chain deposition disease (HCDD) is a rare complication of plasma cell dyscrasia in which monoclonal heavy chains deposit in glomerular and tubular basement membranes of the kidney. Clinical and pathologic features of HCDD have been well described in case reports and series, but evidence supporting specific therapies is sparse. Historically, the disease has had a poor prognosis, intensifying the need to clarify optimal treatments. We describe 3 cases of HCDD with biopsy-proven glomerular involvement, severe nephrotic syndrome, and decline in kidney function that were treated successfully with bortezomib, a proteasome inhibitor. None of these patients had multiple myeloma. In all cases, bortezomib-based therapy resulted in sustained resolution of nephrotic syndrome and improvement in kidney function. All 3 patients developed peripheral neuropathy; otherwise, treatment was well tolerated. To our knowledge, this is the first description of the clinical effectiveness of bortezomib against HCDD.
authors.affiliation: Department of Medicine, Columbia University College of Physicians and Surgeons, New York, NY.
authors.forename: Kinjal
authors.initials: K
authors.lastname: Patel
authors.affiliation: Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN.
authors.forename: John J
authors.initials: JJ
authors.lastname: Dillon
authors.affiliation: Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN; Division of Hematology, Mayo Clinic, Rochester, MN.
authors.forename: Nelson
authors.initials: N
authors.lastname: Leung
authors.affiliation: Division of Nephrology, Columbia University College of Physicians and Surgeons, New York, NY.
authors.forename: Andrew S
authors.initials: AS
authors.lastname: Bomback
authors.affiliation: Division of Nephrology, Columbia University College of Physicians and Surgeons, New York, NY.
authors.forename: Gerald B
authors.initials: GB
authors.lastname: Appel
authors.affiliation: Department of Cell Biology and Pathology, Columbia University College of Physicians and Surgeons, New York, NY.
authors.forename: Vivette
authors.initials: V
authors.lastname: D'Agati
authors.affiliation: Division of Nephrology, Columbia University College of Physicians and Surgeons, New York, NY. Electronic address: pac2004@columbia.edu.
authors.forename: Pietro A
authors.initials: PA
authors.lastname: Canetta
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000494:pharmacology / Q000627:therapeutic use*; D001897:Boronic Acids / Q000494:pharmacology / Q000627:therapeutic use*; D000069286:Bortezomib; D005919:Glomerular Filtration Rate / Q000187:drug effects / Q000502:physiology; D006362:Heavy Chain Disease / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D007143:Immunoglobulin Heavy Chains / Q000378:metabolism; D007668:Kidney / Q000187:drug effects / Q000276:immunology / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D011719:Pyrazines / Q000494:pharmacology / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D001897:Boronic Acids; D007143:Immunoglobulin Heavy Chains; D011719:Pyrazines; D000069286:Bortezomib
keywords: Heavy-chain deposition disease; bortezomib; monoclonal immunoglobulin deposition disease; nephrotic syndrome