New Trends in Nontransplant Therapy for Acquired Aplastic Anemia

Ding, Shaoxue; Fu, Rong

doi:10.2174/1381612828666220418132432

Cited by 1 publication

(1 citation statement)

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“…AA is a rare, life-threatening disease with a reported incidence of 1.6 cases/1,000,000/year [ 3 ]. AA patients are prone to severe infections due to neutropenia, experience bleeding due to thrombocytopenia, and/or fatigue due to anemia [ 4 , 5 ]. AA is associated with higher mortality, especially when pancytopenia becomes severe.…”

Section: Introductionmentioning

confidence: 99%

A Single-Centre Experience of First-Line Romiplostim and Immunosuppressive Therapy in Patients With Aplastic Anemia

Dhingra¹,

Rajoreya²

2023

Cureus

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BackgroundRomiplostim, a thrombopoietin (TPO) receptor antagonist, promotes tri-lineage hematopoiesis in patients with acquired aplastic anemia (AA). However, its efficacy as a first-line treatment in combination with an immunosuppressant, i.e., anti-thymocyte globulin (ATG) and cyclosporine (CSA), remains unexplored. ObjectiveTo assess the efficacy and safety of romiplostim in combination with ATG and CSA as first-line treatment in patients with AA. MethodA single-center, retrospective study of AA patients, where data of patients administered with ATG + CSA + romiplostim as a first-line treatment was included. Romiplostim 5 µg/kg weekly for one month; post that, the dose was increased to 10 µg/kg weekly for the next five months. The primary outcome involves the overall response rate and hematological response at baseline, three months, and six months. ResultData from 12 patients with a median age of 18 years was evaluated. At a median follow-up of six months, 25% achieved a complete response, 41.6% achieved a partial response, and 16.7% had no response. Improvement in tri-lineage hematopoietic response had been seen at six months from baseline, with improvement in absolute neutrophil count (ANC) and platelet count (PC) being the most significant, with an increase of >100% from baseline, followed by total leukocyte count (TLC) (75.13%) and hemoglobin (Hb) (66.07%) from baseline. Two deaths were reported during the treatment. ConclusionRomiplostim, in combination with ATG plus CSA, demonstrated clinically significant outcomes as a firstline treatment in patients with AA. Further studies are required to confirm these findings in larger populations to assess long-term outcomes.

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Section: Introductionmentioning

confidence: 99%