Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy

What Was Tested

This registered phase 2 trial tested Romiplostim for anemia.

Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to IST.

It had two arms (groups): Arm 1: Previously Untreated IST (experimental) and Arm 2: Refractory IST (experimental).

Participants with SAA/vSAA that are previously untreated with IST.

Participants with SAA/vSAA that are refractory to IST.

The registry classes the interventions as drug.

Who Took Part

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Age ≥ 18 years at time of enrollment. Diagnosis of SAA/vSAA confirmed by blood, bone marrow, and cytogenetic studies. An Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1 at screening. Arm 1 only: participant requires initial treatment for SAA/vSAA, no matched related donor is available for allogenic hematopoietic cell transplantation (HCT) and will begin IST with antithymocyte globulin and CsA. Arm 2 only: refractory to at least one course of immunosuppressive therapy including horse or rabbit ATG; or ineligible for ATG treatment and refractory to CsA.

The full record lists 5 inclusion and 6 exclusion criteria; among the exclusions: diagnosed as having congenital aplastic anemia (aa) (fanconi anemia, congenital dyskeratosis, etc); history of other malignancy within the past 5 years, with exceptions; aplastic anemia with hemolytic paroxysmal nocturnal hemoglobinuria (pnh) (hemolytic predominant is defined as lactate dehydrogenase (ldh) \> 1.5 x the upper limit of site normal.

How the Study Was Run

The set-up was non-randomized, parallel-group and open-label.

Groups ran in parallel: each of the anemia participants stayed in one arm throughout.

Being open-label, the Romiplostim study did not mask who received what.

It started in 2023-08-31 with completion listed for 2025-02-25.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Arms 1 and 2: proportion of participants achieving any hematologic response at week 14 (time frame: Week 14).

It also tracked 12 secondary outcomes, for example Arm 1: number of participants who achieve a complete response (CR) or partial response (PR) at week 14, Arms 1 and 2: number of participants with serious adverse events, Arms 1 and 2: change from baseline in Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto (GIMEMA) bleeding scale at week 14, Arms 1 and 2: serum romiplostim trough concentrations, Arms 1 and 2: maximum serum concentration (Cmax) of romiplostim and Arms 1 and 2: area under the curve (AUC) of romiplostim.

What This Means in Plain Language

Put simply, this is a phase 2, non-randomized trial of Romiplostim in people with anemia.

It was withdrawn before enrolling anyone; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Romiplostim compound page, Anemia research.