Favorable response to second-line sirolimus regardless of status in acquired pure red cell aplasia: A retrospective study from a single center.

Sun Z, Jia X, Shen Y et al. · 2026 · Therapeutic Advances in Hematology · Atlas ID SUN2026

What this study shows

Second-line sirolimus achieved a 54.5% overall response rate in ciclosporin-refractory acquired pure red cell aplasia (64.3% in secondary disease) versus 25.0% for first-line ciclosporin, with responses independent of mutation status. Observational: a single-centre retrospective series with no randomisation and no concurrent control arm. The sirolimus and ciclosporin figures come from different lines of therapy in a selected population, so confounding by indication is not excluded and the two rates should not be read as a head-to-head comparison.

Abstract

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Cyclosporine A (CsA) is standard first-line treatment for acquired pure red cell aplasia (PRCA), but outcomes vary and salvage strategies for CsA-refractory patients are poorly defined. Retrospective single-centre comparative study of patients treated 2021-2025: 36 received first-line CsA and 22 switched to sirolimus for lack of response or intolerance. The ORR for first-line CsA was 25.0% (9/36), significantly higher in primary cases (42.1%) than secondary (5.9%). In the salvage sirolimus group ORR was 54.5%, with efficacy in both primary (37.5%) and secondary (64.3%) cases.

Read the full abstract on PubMed →

At a glance

Evidence type	H Human study Marked H because it is direct evidence from a human clinical trial or human cohort; the code names the kind of study, not its quality -- a small, well-run trial is still H.
Study type	3 - Human Observational
Model system	Human (single-centre retrospective, n=58)
Journal	Therapeutic Advances in Hematology
Year	2026
Peer reviewed	Yes
Record last updated	2026-08-22
Source	DOI 10.1177/20406207261483341 · PMID 42643810 · Free full text (PMC13504103)

Extracted findings

Intervention	Sirolimus (second-line salvage)
Target	mTOR
Model	Human
Effect	54.5% ORR in CsA-refractory PRCA; sustained remission in responders; effective regardless of STAT3/5b mutation status

Cite this paper

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Sun, Z., et al. (2026). Favorable response to second-line sirolimus regardless of status in acquired pure red cell aplasia: A retrospective study from a single center. Therapeutic Advances in Hematology. https://doi.org/10.1177/20406207261483341

@article{SUN2026,
  author       = {Sun, Z. and Jia, X. and Shen, Y. and others},
  title        = {{Favorable response to second-line sirolimus regardless of status in acquired pure red cell aplasia: A retrospective study from a single center}},
  journal      = {Therapeutic Advances in Hematology},
  year         = {2026},
  doi          = {10.1177/20406207261483341},
  note         = {PMID: 42643810},
}

Cite this Atlas record

The record is the Atlas's own work — the evidence label, the extracted findings and the links. It is cited as part of the dataset, not as the paper.

Barton, O. (2026). Oliver's mTOR Atlas (record SUN2026) [Data set]. https://mtor-atlas.org/study/SUN2026/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_SUN2026,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record SUN2026},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/SUN2026/},
  doi          = {10.5281/zenodo.22059963}
}