Abstract | PURPOSE: EXPERIMENTAL DESIGN: The FDA reviewed data from two double-blind, placebo-controlled clinical studies, an uncontrolled extension study, and supportive studies. In the controlled studies, enrolled patients had completed at least one prior treatment for chronic ITP and had a platelet count < or = 30 x 10(9)/L. One study enrolled patients who had undergone splenectomy; the other enrolled patients who had not undergone splenectomy. The primary endpoint in both controlled studies was durable platelet response. RESULTS: Overall, 125 patients were randomized in the controlled studies. A durable platelet response was observed in 61% of nonsplenectomized patients and 38% of patients who had undergone splenectomy. One placebo group patient achieved a durable platelet response. Serious hemorrhage events were reported in 10% of placebo recipients and 6% of romiplostim recipients. In the extension study, patients received romiplostim for a median of 60 weeks and a maximum of 96 weeks; the majority of patients maintained platelet counts > or = 50 x 10(9)/L throughout the study. Major safety findings pertained to a risk for bone marrow reticulin formation and worsened thrombocytopenia following romiplostim discontinuation. CONCLUSIONS: The FDA approved romiplostim for use among certain patients with chronic ITP. This approval included a Risk Evaluation and Mitigation Strategy to ensure that the benefits of the drug outweigh its risks.
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Authors | Faranak Jamali, Steven Lemery, Kassa Ayalew, Suzanne Robottom, Kathy Robie-Suh, Dwaine Rieves, Richard Pazdur |
Journal | Oncology (Williston Park, N.Y.)
(Oncology (Williston Park))
Vol. 23
Issue 8
Pg. 704-9
(Jul 2009)
ISSN: 0890-9091 [Print] United States |
PMID | 19711585
(Publication Type: Journal Article, Randomized Controlled Trial)
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Chemical References |
- Carrier Proteins
- Receptors, Fc
- Recombinant Fusion Proteins
- Thrombopoietin
- romiplostim
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Topics |
- Adult
- Aged
- Aged, 80 and over
- Carrier Proteins
(therapeutic use)
- Chronic Disease
- Female
- Humans
- Male
- Middle Aged
- Placebo Effect
- Platelet Count
- Prognosis
- Purpura, Thrombocytopenic, Idiopathic
(drug therapy, surgery)
- Receptors, Fc
(therapeutic use)
- Recombinant Fusion Proteins
(therapeutic use)
- Splenectomy
- Thrombopoietin
- Treatment Outcome
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