A prospective, multicenter study of bortezomib, cyclophosphamide, and dexamethasone in relapsed/refractory iMCD

Abstract

Relapsed and refractory (R/R) idiopathic Multicentric Castleman disease (iMCD) is a clinical challenge with few treatment options. In this first multicenter, prospective trial which implemented the recently published CDCN response criteria, we evaluated the efficacy and safety profiles of bortezomib-cyclophosphamide-dexamethasone (BCD) regimen in 24 R/R iMCD patients. By 6 months, 15 patients (62.5%) achieved overall treatment responses; four patients (16.7%) had stable disease and five patients (20.8%) suffered from progression of disease. Even when considering all patients, there were significant (p < .05) improvements in median symptom score, hemoglobin, platelet count, C-reactive protein (CRP) erythrocyte sedimentation rate (ESR), IL-6, albumin, and immunoglobin G (IgG) after treatment. The regimen was well tolerated without grade 3 or higher adverse events. Estimated 1-year progression-free survival (PFS) and overall survival (OS) were 79% and 92%, respectively. BCD regimen is an effective and safe treatment option for R/R iMCD patients. This trial was registered at www.chictr.org.cn as # ChiCTR1800019342.

Keywords:

• Idiopathic multicentric castleman disease
• bortezomib
• treatment

Author contribution

Contribution: L.Z., X.-x.C., D.-b.Z., Y-j.D., and J.L. recruited the patients; L.Z., Y-j.D., and J.L. designed the study; L.Z., M.-y.Z., Y-j.D., and J.L. collected the data; L.Z. performed the analysis; L.Z., Y-j.D., J.L., and D.C.F. interpreted the data and wrote the manuscript; and all authors had access to primary clinical trial data and gave final approval to submit for publication.

Acknowledgments

The authors thank Ai-lin Zhao for assistance in preparing the graphics.

Disclosure statement

D.C.F has received research funding from Janssen Pharmaceuticals and EUSA Pharma. The remaining authors declare no competing financial interests.

Correction Statement

This article has been corrected with minor changes. These changes do not impact the academic content of the article.

Additional information

Funding

This work was supported by the National Natural Science Foundation of China [81900202 [L.Z.]; Grant No. 81974011 [J.L.]], the Fundamental Research Funds for the Central Universities [3332018036 [L.Z.]], the Capital Characteristic Clinic Project Foundation (Z181100001718206 [Y-j.D]), The CAMS Innovation Fund for Medical Sciences [Grant 2016-12M-1-002 [J.L.]] and The National Key Research and Development Program of China [Grant 2016YFC0901503 [J.L.]] Funding of D.C.F is considered as conflict of interest.