Patient-reported Outcomes for Multicentric Castleman’s Disease in a Randomized, Placebo-controlled Study of Siltuximab

Frits van Rhee, Margaret Rothman, Kai Fai Ho, Sarah Fleming, Raymond S. Wong, Alexander Fosså, Angela Dispenzieri, James Cavet, Nikhil Munshi, Jessica Vermeulen, Corey Casper

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Abstract

Background: Multicentric Castleman’s disease (MCD) is a rare lymphoproliferative disorder driven by dysregulated interleukin-6 production. MCD has a poor prognosis, and treatment is generally noncurative and aimed at symptom relief. Siltuximab is a novel, monoclonal interleukin-6 antibody recently shown to be effective in a registration clinical trial. MCD symptoms, such as fatigue, pain, and weakness, are most appropriately quantified using patient-reported outcome (PRO) measures. We assessed the effect of siltuximab on patient perception of symptoms, functional status, and wellbeing using PRO instruments. Methods: We analyzed results of a randomized, double-blind trial comparing siltuximab 11 mg/kg every 3 weeks with placebo to treat MCD. Subjects (N = 79) completed the recently developed MCD–Symptom Scale (MCD–SS), the Functional Assessment of Chronic Illness Therapy–Fatigue (FACIT–Fatigue) scale, and the Short Form (SF)-36 at predetermined time points throughout the treatment period. Scores were compared at baseline and over time between the treatment arms and PRO instruments. Results: At baseline, the mean number of symptoms reported was 9.2 (standard deviation 3.76) out of 16 total, as measured by the MCD–SS. Fatigue was a key symptom across all PRO instruments. Siltuximab-treated subjects reported early improvements in symptoms compared with subjects in the placebo arm on both the MCD–SS and FACIT–Fatigue scale. Statistically significant improvements in five SF-36 domains were observed in siltuximab-treated patients, namely role physical, role emotional, vitality, bodily pain, and mental health. Conclusions: Patients with MCD commonly report impairments in functioning, wellbeing, and fatigue at baseline. Siltuximab-treated patients reported significant improvements in these outcomes after treatment.

Original languageEnglish (US)
Pages (from-to)207-216
Number of pages10
JournalPatient
Volume8
Issue number2
DOIs
StatePublished - Apr 1 2015

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ASJC Scopus subject areas

  • Nursing (miscellaneous)

Cite this

van Rhee, F., Rothman, M., Ho, K. F., Fleming, S., Wong, R. S., Fosså, A., Dispenzieri, A., Cavet, J., Munshi, N., Vermeulen, J., & Casper, C. (2015). Patient-reported Outcomes for Multicentric Castleman’s Disease in a Randomized, Placebo-controlled Study of Siltuximab. Patient, 8(2), 207-216. https://doi.org/10.1007/s40271-015-0120-5