Corticosteroids plus rhTPO versus corticosteroid monotherapy as initial therapy in elderly patients with newly diagnosed primary immune thrombocytopenia: a propensity score-matched real-world study

Q: What is the clinical evidence from this study?

Study design: Cohort. Population: Newly diagnosed primary immune thrombocytopenia (ITP) (n=124). Intervention: Corticosteroids plus recombinant human thrombopoietin (rhTPO) vs. Corticosteroid monotherapy. Primary outcome: Initial response rate at day 14 (platelet count >= 30 x 10^9/L and at least a 2-fold increase from baseline without bleeding) (p=0.007).

Jia-qi Liu; Lin Sun; Yanyan Xie; Le Jiang; Junjie Xu; Yi-min Zhou; Qing-yang Zhang; Meirong Yang; Shuang Liu; Na Gao; Jian Chen; Liang Wang; Qiaofeng Dong; Yan-ming Wang; Zhi-long Xu; Zhen-yu Yan; Ya‐jing Zhao; Xinguang Liu

doi:10.1186/s12959-026-00938-6

November 5, 2026Thrombosis JournalOpen Access

Corticosteroids plus rhTPO versus corticosteroid monotherapy as initial therapy in elderly patients with newly diagnosed primary immune thrombocytopenia: a propensity score-matched real-world study

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Key result

Adding rhTPO to corticosteroids is linked to ~21 percentage points higher initial response in elderly ITP.

P=0.007
n=124

Why the study?

Elderly patients with primary immune thrombocytopenia are vulnerable to bleeding and corticosteroid toxicity, and corticosteroid monotherapy often fails to achieve durable remission. Whether adding recombinant human thrombopoietin to frontline corticosteroids improves clinical outcomes in these patients needed to be determined.

Does the addition of recombinant human thrombopoietin (rhTPO) to frontline corticosteroids improve initial response and 6-month sustained response compared to corticosteroid monotherapy in elderly patients (≥ 60 years) with newly diagnosed primary immune thrombocytopenia?

Key Points

To determine whether adding recombinant human thrombopoietin (rhTPO) to frontline corticosteroids improves clinical outcomes in elderly patients with newly diagnosed primary immune thrombocytopenia.
Screened patients aged ≥ 60 years with newly diagnosed primary immune thrombocytopenia hospitalized across 10 tertiary hospitals in China between January 2017 and December 2024 (N=175 eligible).
Conducted 1:1 propensity score matching to compare 62 patients receiving corticosteroids plus rhTPO against 62 patients receiving corticosteroid monotherapy, with a median follow-up of 3 months (range: 0.5–96 months).
The combination group showed significantly higher initial response rates (85.5% vs. 64.5%, P = 0.007), complete response rates (59.7% vs. 35.5%, P = 0.007), and 6-month sustained response rates (43.5% vs. 25.8%, P = 0.038), with prolonged duration of response (P = 0.020).
Multivariate logistic regression identified rhTPO as an independent predictor of both initial response (OR = 2.783, 95% CI: 1.210–6.401, P = 0.016) and 6-month sustained response (OR = 2.225, 95% CI: 1.086–4.557, P = 0.029).
Adverse events were mostly grade 1–2 with similar overall incidence (30.6% vs. 43.5%, P = 0.137); thrombotic events occurred in 4 combination patients (6.5%), three of whom had preexisting cardiovascular or cerebrovascular disease.

Study Design

Type

Cohort (n=124)

Multicenter

Yes

Structured PICO

Does the addition of recombinant human thrombopoietin (rhTPO) to frontline corticosteroids improve initial response and 6-month sustained response compared to corticosteroid monotherapy in elderly patients (≥ 60 years) with newly diagnosed primary immune thrombocytopenia?

P

Population

124 propensity score-matched hospitalized older adults (aged 60-84 years; 55.6% female) with newly diagnosed primary immune thrombocytopenia across 10 centers in China, followed for a median of 3 months (range 0.5-96 months).

E

Exposure

Frontline corticosteroids (high-dose dexamethasone 40 mg/day for 4 days [1–2 cycles], prednisone 1 mg/kg/day, or methylprednisolone 1–2 mg/kg/day, tapered and discontinued within 8 weeks) combined with recombinant human thrombopoietin (rhTPO) initiated within 7 days of corticosteroid treatment at 300–600 U/kg/day for 5 to 25 days (post-PSM). Short-term IVIg, platelet transfusion, and hemostatic agents were permitted when clinically indicated.

C

Comparator

Frontline corticosteroid monotherapy (high-dose dexamethasone 40 mg/day for 4 days [1–2 cycles], prednisone 1 mg/kg/day, or methylprednisolone 1–2 mg/kg/day, tapered and discontinued within 8 weeks), with short-term IVIg, platelet transfusion, and hemostatic agents permitted when clinically indicated.

O

Outcome

Initial response at day 14 (defined as complete response [platelet count ≥ 100 × 10^9/L without bleeding] or response [platelet count ≥ 30 × 10^9/L and at least a 2-fold increase from baseline without bleeding], confirmed on at least 2 occasions ≥ 7 days apart) and 6-month sustained response (maintenance of CR or R for ≥ 6 months without additional ITP-specific treatments).surrogate

Main Result

Absolute Event Rate: 85.5% vs 64.5%

p-value: p=0.007

Adding recombinant human thrombopoietin (rhTPO) to frontline corticosteroids significantly improves initial and sustained platelet responses in elderly patients with newly diagnosed primary immune thrombocytopenia, though caution is warranted in patients with preexisting cardiovascular or cerebrovascular disease due to a numerically higher rate of thrombotic events.

Limitations

Retrospective design with potential selection and information bias despite propensity score matching
Residual confounding from unmeasured variables
Heterogeneous corticosteroid regimens (dexamethasone, prednisone, and methylprednisolone) and variations in rhTPO dosing and duration
Underpowered subgroup analyses and small sample size limiting definitive conclusions regarding thrombotic risk
Relatively small sample sizes in corticosteroid regimen subgroups, requiring cautious interpretation of subgroup findings
Non-randomized retrospective observational design requiring prospective randomized trials with predefined thrombotic surveillance to confirm efficacy and safety

Ask about this paper

Answers are grounded in this paper and cite their sources.

Implication

May support rhTPO addition in elderly ITP; leaves open confirmation by randomized trials before practice change.

Abstract

Background Elderly patients with primary immune thrombocytopenia (ITP) are particularly vulnerable to bleeding and corticosteroid-related toxicity, and management is often complicated by comorbidities. Corticosteroid monotherapy often fails to achieve durable remission, underscoring the need for more effective combination strategies. Objective To determine whether the addition of recombinant human thrombopoietin (rhTPO) to frontline corticosteroids improves clinical outcomes in elderly patients with newly diagnosed ITP. Methods Patients aged ≥ 60 years with newly diagnosed ITP hospitalized at 10 tertiary hospitals in China between January 2017 and December 2024 were screened. After 1:1 propensity score matching, 62 patients remained in each treatment group: corticosteroids plus rhTPO and corticosteroid monotherapy group. Multivariate logistic regression was conducted to identify independent predictors of initial response and 6-month sustained response (SR). Kaplan-Meier analysis was employed to assess duration of response (DOR). Results Compared with corticosteroid monotherapy, combination group demonstrated significantly higher rates of initial response (85.5% vs . 64.5%, P = 0.007), CR (59.7% vs . 35.5%, P = 0.007), and 6-month SR (43.5% vs . 25.8%, P = 0.038), along with prolonged DOR ( P = 0.020). Multivariate logistic regression analysis identified rhTPO use as an independent predictor of both initial response and 6-month SR. Adverse events were predominantly grade 1–2 and occurred at comparable frequencies between groups. Thrombotic events were numerically higher in the combination cohort and were mainly observed in patients with preexisting cardiovascular or cerebrovascular disease, although statistical significance was not reached. Conclusions Frontline corticosteroids plus rhTPO therapy was associated with improved clinical outcomes in elderly patients with newly diagnosed ITP while maintaining a good safety profile. Prospective randomized trials with predefined thrombotic surveillance are warranted to confirm therapeutic efficacy and fully characterize safety.

Discussion

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Cite This Study

Liu et al. (2026) conducted a cohort in Newly diagnosed primary immune thrombocytopenia (ITP) (n=124). Corticosteroids plus recombinant human thrombopoietin (rhTPO) vs. Corticosteroid monotherapy was evaluated on Initial response rate at day 14 (platelet count >= 30 x 10^9/L and at least a 2-fold increase from baseline without bleeding) (p=0.007). Corticosteroids plus rhTPO increased initial response rates (85.5% vs 64.5%, P=0.007) and 6-month sustained response (43.5% vs 25.8%, P=0.038) compared to steroid monotherapy in elderly ITP patients.

synapsesocial.com/papers/6aee6b6871a75948062208c6 https://doi.org/10.1186/s12959-026-00938-6

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Corticosteroids plus rhTPO versus corticosteroid monotherapy as initial therapy in elderly patients with newly diagnosed primary immune thrombocytopenia: a propensity score-matched real-world study

Key Points

Study Design

Structured PICO

Main Result

Limitations

Ask about this paper

Implication

Abstract

Discussion

Cite This Study

Corticosteroids plus rhTPO versus corticosteroid monotherapy as initial therapy in elderly patients with newly diagnosed primary immune thrombocytopenia: a propensity score-matched real-world study

Key Points

Study Design

Structured PICO

Main Result

Limitations

Ask about this paper

Implication

Abstract

Discussion

Cite This Study

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