High- vs regular-dose recombinant human thrombopoietin plus cyclosporine A in patients with newly diagnosed non-severe aplastic anemia: a retrospective cohort study

ABSTRACT

Background:

Cyclosporine A (CsA) and regular doses of recombinant human thrombopoietin (rhTPO) can accelerate platelet recovery in patients with non-severe aplastic anemia (NSAA). However, it is unclear whether CsA plus rhTPO at a higher dose can further increase the efficacy.

Methods:

Data from patients with newly diagnosed NSAA, who had been treated with CsA in combination with different doses of rhTPO between February 2021 and August 2021 at Peking Union Medical College Hospital, were reviewed. All the enrolled patients had been treated with CsA at 3–5 mg/(kg/d), and patients were further classified into high-dose (with rhTPO 30000U qd × 14 days for 2 months) group or regular-dose (with rhTPO 15000U qd × 7days for 3 months) group. The treatment response and therapy-related adverse events were compared.

Results:

36 patients including 16 (44.4%) in the high-dose and 20 (55.6%) in the regular-dose group were enrolled. The baseline characteristics were compatible between the two groups. The platelet counts were significantly higher at 1/3/6 months in the high-dose group (p = 0.028, 0.0063 and p = 0.040, respectively). The high-dose group had a significantly shorter time to platelet transfusion independence ([1 (0.5–6) months vs 2.5 (1–12) months, p = 0.040]). There was no significant difference in overall response and complete response rate between the two groups at 1/3/6/12 months (p > 0.05). Treatment-related morbidities were similar between the two groups (p > 0.05).

Conclusions:

Adding a higher dose of rhTPO can further accelerate platelet recovery and platelet transfusion independence in patients with newly diagnosed NSAA.

KEYWORDS:

• Non-severe aplastic anemia
• recombinant human thrombopoietin
• cyclosporine A
• response
• platelet recovery

Acknowledgements

We would like to thank all the patients who participated in this study and their supportive families, as well as the investigators and clinical research staff from our centers.

Disclosure statement

No potential conflict of interest was reported by the author(s).

Data availability statement

All data are available in the main text. All detailed metadata are available upon reasonable request to the corresponding author.

Author contributions

Yuan Yang and Bing Han: study design and study concept. Yuan Yang, Qinglin Hu: data collection. Yuan Yang and Qinglin Hu: statistical analysis, data visualization. Yuan Yang: manuscript drafting. Yuan Yang, Chen Yang, Miao Chen, and Bing Han: data interpretation.

Consent for publication

The publication of this manuscript has been approved by all authors.

Additional information

Funding

This retrospective study was supported by fundings from CAMS Innovation Fund for Medical Sciences (CIFMS 2021-I2M-1-003), National High Level Hospital Clinical Research Funding (2022-PUMCH-C-026, 2022-PUMCH-D-002, 2022-PUMCH-B-046). The funders played no roles in the study design, the collection, analysis, and interpretation of data, the writing of the report, and the decision to submit the article for publication. All authors have no financial or non-financial conflicts to disclose in this study.