Safety and efficacy of self-administered romiplostim in patients with immune thrombocytopenia: Results of an integrated database of five clinical trials

Abstract

Romiplostim self-administration by patients or caregivers may offer time/cost savings to healthcare professionals (HCPs) and convenience for patients who avoid weekly clinic visits. We performed an integrated analysis of five clinical trials to evaluate the efficacy and safety of romiplostim self-administration. Data were analyzed from adults with immune thrombocytopenia (ITP) who received weekly romiplostim via self-administration or from an HCP. Patients who achieved a stable romiplostim dose for ≥3 weeks (HCP group ≥5 weeks to provide an appropriate index date to enable comparisons with the self-administration group) with platelet counts ≥50 × 109 /L were eligible. In the self-administration (n = 621) vs HCP (n = 133) groups, respectively, median age was 53 vs 58 years, median time since primary ITP diagnosis was 3.7 vs 2.5 years, and median baseline platelet count at ITP diagnosis was 19.0 vs 20.0 × 109 /L. In the self-administration and HCP-dosed groups, median romiplostim treatment duration was 89 vs 52 weeks and median total number of doses was 81 vs 50, respectively. In the self-administration and HCP groups, respectively: 95.0% and 100.0% of patients achieved ≥1 platelet response (defined as weekly platelet count ≥50 × 109 /L without rescue medication in previous 4 weeks); the median percentage of weeks with a response was 94.5% and 95.9%; and rescue medication was used in 36.7% and 39.8% of patients. Self-administration did not adversely affect safety; duration-adjusted rates for all treatment-emergent adverse events (TEAEs) and bleeding TEAEs were numerically lower with self-administration. Romiplostim self-administration appears effective and well tolerated in eligible patients with ITP.

Conflict of interest statement

D.J.K. has received research funding from Actelion (Syntimmune), Agios, Alnylam, Amgen, Argenx, BristolMyers Squibb, Kezar, Principia, Protalex, Rigel, Takeda (Bioverativ), and UCB, and acted as a consultant for Actelion (Syntimmune), Agios, Alnylam, Amgen, Argenx, BristolMyers Squibb, Caremark, Daiichi Sankyo, Dova, Kyowa‐Kirin, Merck, Sharp & Dohme, Momenta, Novartis, Pfizer, Platelet Disorder Support Association, Principia, Protalex, Protalix, Rigel, Rubius, Sanofi, Genzyme, Shionogi, Shire, Takeda (Bioverativ), UCB, Up‐To‐Date, and Zafgen. D.M.A. has received research funding from Novartis and BristolMyers Squibb, and acted as a consultant for Rigel, Principia, Novartis, and Amgen. F.R. has acted as a consultant for, received lecture fees from, and received research funding from Amgen and Novartis. A.J. has participated on advisory boards, and received travel grants and speakerʼs fees for AbbVie, Amgen, Celgene, Janssen, Novartis, Roche, and Sanofi Genzyme. D.S. has acted as a consultant for and participated in speakerʼs bureau for Amgen. R.B. has been part of a speaker faculty (honoraria declined) for Amgen and Novartis, and acted as a principal investigator in clinical trials for Amgen, Novartis, CSL, BristolMyers Squibb, Principia Pharma, and Rigel. A.N. has acted as a consultant for Amgen, Angle, Argenx, Dova, Novartis, Ono, Rigel, and Shionogi, received research funding from Amgen, Novartis, and Rigel, received honoraria from Amgen, Angle, Argenx, Dova, Novartis, Ono, Rigel, and Shionogi, and provided paid expert testimony for Argenx and Rigel. J.M. has received research funding from Amgen, Dova Pharmaceuticals and Rigel. K.M. and R.O. are employed by Amgen.

© 2020 The Authors. American Journal of Hematology published by Wiley Periodicals, Inc.

Figures

Figure 1

A, Proportions of patients in platelet count categories immediately prior to (Weeks −4 to −1) and during the study period; *, B, Rescue medication requirements by study period. *Patients in the self‐administration group were required to have ≥3 consecutive stable doses (maintaining platelet counts at ≥50 × 109/L) ‐ therefore, during Weeks −4 to –1 some patients in the self‐administration group may have had counts <50 × 109/L on one of their visits. HCP, healthcare professional dosing; SA, self‐administration