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Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial
Plain-language guide to PMID 31474546 (2019): open-label randomized controlled trial study of Romiplostim.
Key Facts
- PMID
- 31474546
- Journal
- The Lancet. Haematology
- Year
- 2019
- Study type
- Randomized Controlled Trial
- Authors
- Lee JW et al. (9 authors)
- DOI
- 10.1016/S2352-3026(19)30153-X
What This Study Did, in Plain Language
What Was Tested
Researchers studied Romiplostim in this randomized controlled trial publication.
In this phase 2 trial the researchers aimed to assess the activity and safety of romiplostim in patients with aplastic anaemia who were previously treated with immunosuppressive therapy.
Why It Was Studied
Aplastic anaemia is a rare, life-threatening condition, characterised by pancytopenia with hypocellular bone marrow.
Haematopoietic stem cells and most progenitor cells express thrombopoietin receptor (c-MPL).
Who Took Part
The age groups tagged for this paper are adults and middle-aged adults, both men and women.
Eligible patients were aged 19 years or older, and had aplastic anaemia confirmed by bone marrow and cytogenetic studies and thrombocytopenia (platelet count ≤30 × 109/L), an Eastern Cooperative Oncology Group performance status score of 2 or lower, and were previously treated with immunosuppressive therapy, including at least one course of antithymocyte globulin plus cyclosporin.
How the Study Was Run
The design is described as randomized, open-label and parallel-group.
The participants were allocated by chance, so the Romiplostim group and the comparison group should start out alike.
Being open-label, the Romiplostim study did not mask who received what.
The Romiplostim group and the comparison group ran side by side rather than switching.
This trial is registered with ClinicalTrials.gov, NCT02094417.
What Was Measured
The subject headings attached to it are Anemia, Aplastic, Blood Platelets, Immunosuppressive Agents, Myalgia, Neutrophils, Receptors, Fc and Recombinant Fusion Proteins.
What This Means in Plain Language
The paper is a randomized, open-label and parallel-group study of Romiplostim.
Results are reported in around 10 sentences of the abstract; we leave them to the paper itself rather than summarise them.
The publication is from 2019 (The Lancet. Haematology), a journal based in England.
More context: the Romiplostim compound page, all human trials.
Our Notes on the Compound
Romiplostim: A peptide-antibody fusion (peptibody) that activates the thrombopoietin receptor to raise platelet counts; sold as Nplate.
Related Pages
Source: PubMed record. This explanation is written by PeptideWhiz from the public record. For research and education only. Not medical advice; no dosing information is given.