Observational studyLeukemia2023
Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study.
Observational study in Leukemia, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled 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.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
16 citing papers in PubMed, 1 synthesis or guideline pooled it, 15 citations in OpenAlex.
- American Society of Hematology 2026 guidelines for the diagnosis and management of severe and very severe acquired aplastic anemia.Blood advances · 2026Guideline
- Trial
- Hetrombopag Added to Cyclosporine as the First-Line Treatment for Patients With Non-Severe Aplastic Anemia: A Phase 2 Multicenter Trial.American journal of hematology · 2026Trial
- Efficacy of ciclosporin monotherapy in non-severe aplastic anaemia not requiring transfusions: Results from a multicentre phase II study.British journal of haematology · 2026Trial
- Evidence- and opinion-based consensus from Delphi round 1 of the Asian aplastic anemia recommendation development.International journal of hematology · 2026Article
- Safety, pharmacokinetics, and preliminary efficacy of hetrombopag in pediatric patients with aplastic anemia: A pilot study.Annals of hematology · 2026Article
- Cyclosporine in hematological disorders: mechanisms, clinical practice and emerging advances.Annals of hematology · 2026Review
- Development and validation of a nomogram for early prognostic prediction in childhood aplastic anemia receiving cyclosporine monotherapy.Pediatric research · 2026Article
- Machine learning mortality prediction model for cyclosporine therapy in pediatric aplastic anemia.Annals of hematology · 2026Article
- Efficacy and safety of different TPO-RAs combined with cyclosporine in the treatment of NSAA.Therapeutic advances in hematology · 2026Article
- Macrophages in Hematopoiesis and Related Blood Diseases.Genomics, proteomics & bioinformatics · 2025Review
- Elderly Patients With Aplastic Anemia: Treatment Patterns and Outcomes in the Real World.American journal of hematology · 2025Article
- Cyclosporine plus avatrombopag versus cyclosporine monotherapy for first-line treatment of elderly patients with transfusion-dependent non-severe aplastic anemia: a single center retrospective study.Annals of medicine · 2024Article
- Monitoring and Treatment of Paroxysmal Nocturnal Hemoglobinuria in Patients with Aplastic Anemia in Asia: An Expert Consensus.International journal of molecular sciences · 2024Review
- Diagnostic evaluation in bone marrow failure disorders: what have we learnt to help inform the transplant decision in 2024 and beyond?Bone marrow transplantation · 2024Review
- Aberrant myelomonocytic CD56 expression predicts response to cyclosporine therapy in pediatric patients with moderate aplastic anemia.Frontiers in pediatrics · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors at 7 institutions in 3 countries.
Funding
Abstract
Non-severe aplastic anemia is a rare bone marrow failure disorder characterized by variable degrees and combination of cytopenias, with limited data on management and outcome. We describe a large multicentric series of 259 patients, focusing on clinical and molecular features, treatment, evolution, and survival. The majority required treatment with cyclosporine (CyA) alone (N = 84) or in combination with anti-thymocyte globulin (ATG,44) or eltrombopag (20), eltrombopag alone (10), or others (25) including androgens. Similar outcomes were observed across different strategies, with a 6-month overall response rate of 73% for CyA, 74% for ATG plus CyA, 68% for CyA plus eltrombopag, 87% for eltrombopag, and 79% for others. Notably, 56 patients (39%), mainly receiving CyA plus eltrombopag, achieved a trilineage response (p = 0.02). Progression to myeloid neoplasms was limited (8%) and not related to mutational status. Hemolytic PNH developed in 10% of cases, being predicted by detection of small clones at diagnosis. Survival was negatively impacted by age, male gender, LDH, platelets/erythrocyte transfusion need, and somatic mutations by NGS, and positively by higher neutrophils at diagnosis, PNH clones, and trilineage response at 6 and 12 months. Multivariable analysis confirmed the detrimental role of age and the favorable association with PNH clone and trilineage response at 6 months.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.