Belimumab and voclosporin for lupus nephritis: effectiveness and value : final report

Belimumab and voclosporin for lupus nephritis: effectiveness and value : final report

Collection:
Health Policy and Services Research
Author(s):
Tice, Jeffrey A., author
Agboola, Foluso, author
Herron-Smith, Serina, author
Chapman, Rick, (Of Institute for Clinical and Economic Review), author
Pearson, Steven D., author
Mandrik, Olena, author
Thokala, P. (Praveen), author
Fotheringham, James, (Of University of Sheffield), author
Contributor(s):
Institute for Clinical and Economic Review, issuing body.
New England Comparative Effectiveness Public Advisory Council, issuing body.
Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, April 16, 2021
Language(s):
English
Format:
Text
Subject(s):
Antibodies, Monoclonal, Humanized
B-Cell Activating Factor -- antagonists & inhibitors
Calcineurin Inhibitors -- therapeutic use
Cyclosporine -- therapeutic use
Immunosuppressive Agents -- therapeutic use
Lupus Nephritis -- drug therapy
Treatment Outcome
United States
Genre(s):
Technical Report
Abstract:
Systemic lupus erythematosus (SLE) is an autoimmune disease that affects between 300,000 and 1.5 million Americans. It is more common in women (90% of diagnosed cases) and in non-Whites (four times higher prevalence in Black patients, two times higher prevalence in Hispanic patients). Approximately half of patients with SLE will be diagnosed with lupus nephritis (LN), characterized by inflammation in the kidney, proteinuria, and progressive kidney damage which can lead to kidney failure. LN typically presents in patients who are 20 to 40 years old and is the most common cause of death and disability in patients with SLE. In this report, ICER reviews belimumab, a parenteral b-lymphocyte inhibitor, and voclosporin, an oral calcineurin inhibitor, for the initial treatment of patients with LN. Each drug is added to standard induction therapy for LN which is high-dose corticosteroids combined with either mycophenolate mofetil (MMF) or cyclophosphamide. The FDA approved belimumab on 12/17/2020 and voclosporin on 01/22/2021. Belimumab increases the complete renal response (CRR) and primary efficacy renal response (PERR) at two years compared with standard therapy alone, with benefits seen after the first year appearing stable at year two. At two years, the proportion of patients receiving ≤ 5 mg of prednisone was greater in the belimumab group (36.8% versus 27.8%). There were no significant increases in adverse events or discontinuations compared with standard induction therapy for LN. Voclosporin nearly doubled the complete response (CR) and markedly increased the partial response (PR) at one year compared with standard therapy alone. The proportion of patients on low dose steroids was not reported, but all those with PR and CR were required to be taking low dose steroids. Adverse events were comparable to standard induction therapy for LN, but the FDA added a black box warning consistent with that of cyclosporin for possible serious infections and malignancies. The most important uncertainty is how these short-term assessments of renal response translate into meaningful long-term outcomes for patients in whom SLE is a lifetime illness. In addition, the length of time these therapies are used prior to tapering them to standard maintenance therapy remains to be established. Because of inadequate representation of patients from communities of color in the development trials, the limited data available are highly uncertain and cannot be used to determine the relative effectiveness of either drug among different racial and ethnic groups.
Copyright:
Reproduced with permission of the copyright holder. Further use of the material is subject to CC BY license. (More information)
Extent:
1 online resource (1 PDF file (vi, ES4, 155 pages))
Illustrations:
Illustrations
NLM Unique ID:
9918680386806676 (See catalog record)
Permanent Link:
http://resource.nlm.nih.gov/9918680386806676