Platelet transfusion refractoriness (PTR), a life-threatening clinical complication. TPO-RAs have been widely used to treat thrombocytopenia in various diseases. No studies have reported the treatment of TPO-RAs in refractory PTR. Therefore, we conducted a retrospective study to evaluate the efficacy and safety of TPO-RAs in patients with refractory PTR.

A total of 55 patients with refractory PTR from April 2017 to Feb 2022 were enrolled in Southern Hospital Affiliated with Southern Medical University. We defined refractory PTR as the CCI less than 5 × 109/L after transfusion of two consecutive rounds of immunoglobulin or HLA- matched platelet. 24 patients in the control group received the best available therapies (BATs), such as IVIG、HLA-matched platelet transfusions or steroids. 31 patients in the treatment group received TPO-RAs. TPO-RAs includes Eltrombopag or Avatrombopag.

Platelet recovery (PR) was defined as platelet recovery to ≥50 ×109/L without platelet transfusion support for 7 consecutive days. In the treatment and control groups, the cumulative incidence of PR within 3-months after treatment was respectively 61.3% and 12.5 % (P = .000). On days 7, 14, 21, and 28 after treatment, significantly higher median platelet counts were observed in the treatment group than those in the control group [(24.5 ± 37.10) ×109 /L vs [(10.0 ± 8.58) ×109/L, P=.001; (32.5 ± 62.63) ×109/L vs. (15.0 ±33.28) ×109/L, P=.025; (82.0 ± 103.66) ×109/L vs. (16.0 ± 96.32) ×109/L, P=.000; and (138.0 ± 134.04) ×109/L vs. (18.0 ± 40.74) ×109/L, P=.000 respectively]. The rate of platelet-transfusion independence on day 28 was 70.1% vs.37.5% (treatment group vs. control group, P=.000); the median cumulative platelet transfusion units were 10.0 vs 14.0 respectively (P=0.008). Compared with the control group, the incidence of any bleeding event in the treatment group was lower (83.3% vs 25.8%, P < .001), and the total survival rate increased, 64.5% and 33.3% (P = .000). No patient stopped using TPO-RAs due to side effect or intolerability.

In conclusion, our research demonstrates that TPO-RAs are beneficial and secure for refractory PTR patients. Time to achieved PR is approximately 14 days.

No relevant conflicts of interest to declare.

Author notes

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Asterisk with author names denotes non-ASH members.

© 2022 by The American Society of Hematology
2022
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