Terrell et al

Background Romiplostim increases platelet counts in ITP and is typically injected at clinic visits.

Objective To estimate the efficacy and safety of romiplostim self-administration, we evaluated data from an open-label extension study in a post hoc analysis.

Methods Patients received weekly romiplostim with dose adjustments to target platelet counts of 50-200 x 10⁹/L. Patients with a stable dose and platelet counts of 50-200 x 10⁹/L for 3 or more weeks could begin self-administration if investigators deemed it appropriate, returning to study sites every 4 weeks.

Results Of 292 patients, 239 (82%) initiated self-administration for a median of 74 (Q1-Q3:56-164) weeks. Twenty-eight of the 239 (12%) discontinued self-administration (investigator or sponsor decision: 19, patient request: 6, noncompliance: 3). The median average weekly dose for patients self-administering romiplostim was 4.1  g/kg. The romiplostim dose was adjusted in 40 (17%) of the 239 patients in the first 8 weeks of self-administration; 84 (35%) in the first 6 months. Patients had a platelet response (more than 50 x 10⁹/L) for a mean of 75.1% of weeks. The adverse event (AE) rate was 18.3/100 patient-weeks, with 0.8 serious AEs/100 patient-weeks. Fourteen AEs led to withdrawal; none related to self-administration.

Limitations The analysis was post hoc. Lack of a randomized comparator group may have resulted in differences between patient populations. No distinctions could be made between constant and intermittent self-administration or between adverse events occurring during self-administration or administration at the study site.

Conclusions Patients were able to maintain platelet responses for a mean of 75% of the time without new safety issues while self-administering romiplostim.

To read the full article, click on the PDF icon at the top of this introduction.

Background Romiplostim increases platelet counts in ITP and is typically injected at clinic visits.

Objective To estimate the efficacy and safety of romiplostim self-administration, we evaluated data from an open-label extension study in a post hoc analysis.

Methods Patients received weekly romiplostim with dose adjustments to target platelet counts of 50-200 x 10⁹/L. Patients with a stable dose and platelet counts of 50-200 x 10⁹/L for 3 or more weeks could begin self-administration if investigators deemed it appropriate, returning to study sites every 4 weeks.

Results Of 292 patients, 239 (82%) initiated self-administration for a median of 74 (Q1-Q3:56-164) weeks. Twenty-eight of the 239 (12%) discontinued self-administration (investigator or sponsor decision: 19, patient request: 6, noncompliance: 3). The median average weekly dose for patients self-administering romiplostim was 4.1  g/kg. The romiplostim dose was adjusted in 40 (17%) of the 239 patients in the first 8 weeks of self-administration; 84 (35%) in the first 6 months. Patients had a platelet response (more than 50 x 10⁹/L) for a mean of 75.1% of weeks. The adverse event (AE) rate was 18.3/100 patient-weeks, with 0.8 serious AEs/100 patient-weeks. Fourteen AEs led to withdrawal; none related to self-administration.

Limitations The analysis was post hoc. Lack of a randomized comparator group may have resulted in differences between patient populations. No distinctions could be made between constant and intermittent self-administration or between adverse events occurring during self-administration or administration at the study site.

Conclusions Patients were able to maintain platelet responses for a mean of 75% of the time without new safety issues while self-administering romiplostim.

To read the full article, click on the PDF icon at the top of this introduction.

Background Romiplostim increases platelet counts in ITP and is typically injected at clinic visits.

Objective To estimate the efficacy and safety of romiplostim self-administration, we evaluated data from an open-label extension study in a post hoc analysis.

Methods Patients received weekly romiplostim with dose adjustments to target platelet counts of 50-200 x 10⁹/L. Patients with a stable dose and platelet counts of 50-200 x 10⁹/L for 3 or more weeks could begin self-administration if investigators deemed it appropriate, returning to study sites every 4 weeks.

Results Of 292 patients, 239 (82%) initiated self-administration for a median of 74 (Q1-Q3:56-164) weeks. Twenty-eight of the 239 (12%) discontinued self-administration (investigator or sponsor decision: 19, patient request: 6, noncompliance: 3). The median average weekly dose for patients self-administering romiplostim was 4.1  g/kg. The romiplostim dose was adjusted in 40 (17%) of the 239 patients in the first 8 weeks of self-administration; 84 (35%) in the first 6 months. Patients had a platelet response (more than 50 x 10⁹/L) for a mean of 75.1% of weeks. The adverse event (AE) rate was 18.3/100 patient-weeks, with 0.8 serious AEs/100 patient-weeks. Fourteen AEs led to withdrawal; none related to self-administration.

Limitations The analysis was post hoc. Lack of a randomized comparator group may have resulted in differences between patient populations. No distinctions could be made between constant and intermittent self-administration or between adverse events occurring during self-administration or administration at the study site.

Conclusions Patients were able to maintain platelet responses for a mean of 75% of the time without new safety issues while self-administering romiplostim.

To read the full article, click on the PDF icon at the top of this introduction.