# Robust predictors for drug response of patients with acute myeloid leukemia

**Authors:** Bahar Tercan

PMC · DOI: 10.1371/journal.pone.0343422 · PLOS One · 2026-02-23

## TL;DR

This paper introduces a new method for predicting drug responses in acute myeloid leukemia patients that works well even with imbalanced data.

## Contribution

The novel k-Top Scoring Pairs (kTSP) classifier outperforms existing methods in drug response prediction for AML patients.

## Key findings

- kTSP classifiers perform better than other methods when patient groups are imbalanced.
- The kTSP method is robust to batch effects and suitable for single-patient classification.
- The rank-based approach of kTSP provides reliable drug response predictions for AML.

## Abstract

The significant heterogeneity in treatment responses among patients with acute myeloid leukemia (AML) underscores the critical need for accurate drug response prediction. We developed k-Top Scoring Pairs (kTSP) classifiers, ensemble methods that aggregate the relative expression of gene pairs. We compared their accuracy with that of state-of-the-art machine learning methods, linear and radial basis function support vector machines, random forest and elastic net regression classifiers for drug response prediction of patients with AML. Our results demonstrate that kTSP particularly outperforms other methods when the number of sensitive and resistant patients is imbalanced, a common challenge in clinical studies. Our approach is inherently robust to batch effects and uniquely suited for single-patient classification due to its rank-based methodology.

## Linked entities

- **Diseases:** acute myeloid leukemia (MONDO:0015667)

## Full-text entities

- **Genes:** NPM1 (nucleophosmin 1) [NCBI Gene 4869] {aka B23, NPM}, CD33 (CD33 molecule) [NCBI Gene 945] {aka CD33rSiglec, SIGLEC-3, SIGLEC3, p67}, LILRB1 (leukocyte immunoglobulin like receptor B1) [NCBI Gene 10859] {aka CD85J, ILT-2, ILT2, LIR-1, LIR1, MIR-7}, IDH1 (isocitrate dehydrogenase (NADP(+)) 1) [NCBI Gene 3417] {aka HEL-216, HEL-S-26, IDCD, IDH, IDP, IDPC}, DNER (delta/notch like EGF repeat containing) [NCBI Gene 92737] {aka UNQ26, bet}, BCL2 (BCL2 apoptosis regulator) [NCBI Gene 596] {aka Bcl-2, PPP1R50}, PCBP3 (poly(rC) binding protein 3) [NCBI Gene 54039] {aka ALPHA-CP3, PCBP3-OT1, PCBP3OT}, FLT3 (fms related receptor tyrosine kinase 3) [NCBI Gene 2322] {aka CD135, FLK-2, FLK2, STK1}, CYSLTR2 (cysteinyl leukotriene receptor 2) [NCBI Gene 57105] {aka CYSLT2, CYSLT2R, GPCR21, HG57, HPN321, KPG_011}
- **Diseases:** bone marrow failure (MESH:D000080983), AML (MESH:D015470), hematologic malignancies (MESH:D019337), MDS (MESH:D009190), MPN (MESH:D009369), myeloid disorder (MESH:D007951), inflammatory (MESH:D007249)
- **Chemicals:** midostaurin (MESH:C059539), cytarabine (MESH:D003561), quizartinib (MESH:C544967), cysteinyl leukotrienes (MESH:C112381), Sorafenib (MESH:D000077157), GO (MESH:D000079982), daunorubicin (MESH:D003630), Venetoclax (MESH:C579720)
- **Species:** Homo sapiens (human, species) [taxon 9606]

## Full text

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## Figures

5 figures with captions in the complete paper: https://tomesphere.com/paper/PMC12928390/full.md

## References

42 references — full list in the complete paper: https://tomesphere.com/paper/PMC12928390/full.md

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Source: https://tomesphere.com/paper/PMC12928390