Evidence map›Paper›PMID 42162278›Full record

ArticlePediatric research2026

Superior efficacy of CsA plus ATG over CsA monotherapy in pediatric transfusion-independent moderate aplastic anemia.

Hongmin Li, Lingling Fu, Bixi Yang, Ruixin Wang, Hui Chen, Jie Ma

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Article in Pediatric research, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

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Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

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PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Hongmin LiDepartment of Hematology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Lingling FuDepartment of Hematology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Bixi YangDepartment of Hematology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Ruixin WangDepartment of Hematology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Hui ChenDepartment of Clinical Laboratory Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Jie MaDepartment of Hematology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China. jie_ma2023@163.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundData regarding the management of transfusion-independent moderate aplastic anemia (MAA) in pediatric patients remains scarce. This study was conducted to address this research gap.

methodsWe performed a prospective randomized controlled trial enrolling 29 children with transfusion-independent MAA. Patients were randomly assigned to receive either cyclosporine (CsA) monotherapy (n = 14) or CsA combined with anti-thymocyte globulin (ATG) (n = 15).

resultsBaseline characteristics were well balanced between the two groups. The CsA+ATG group exhibited a higher overall response rate (ORR: 80% vs. 50%) and a significantly higher complete response rate (CRR: 60% vs. 7.1%, P = 0.005) compared with the CsA monotherapy group. At 12 months post-treatment, the combination group demonstrated better hematologic recovery. The 36-month event-free survival (EFS) rate was higher in the combination group than in the monotherapy group (86.7% ± 8.8% vs. 32.5% ± 18.5%), though this difference did not reach statistical significance (P > 0.05), likely due to the small sample size. Shorter pre-treatment disease duration correlated with better response (P = 0.045). Adverse events in both groups were mild to moderate and clinically manageable.

conclusionCsA combined with ATG is superior to CsA monotherapy for pediatric patients with transfusion-independent MAA, with a favorable safety profile. Early initiation of immunosuppressive therapy (IST) within 3 months of diagnosis may correlate with better treatment response. IMPACT: In this study, 29 pediatric patients with transfusion-independent MAA were randomly assigned to two treatment arms (CsA monotherapy, n = 14; CsA+ATG, n = 15). Key findings include: (1) a significantly higher CRR in the combination group (60% vs. 7.1%, P = 0.005); (2) a more than two-fold higher 36-month EFS rate in the combination group (86.7% vs. 32.5%); (3) mild-to-moderate, manageable adverse events in both groups. (4) Shorter pre-treatment disease duration was associated with favorable treatment response (P = 0.045). This study fills the gap in the treatment of transfusion-independent MAA and provides a basis for clinical decision-making.

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PMID42162278

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.