PMID- 27091471 OWN - NLM STAT- MEDLINE DCOM- 20170309 LR - 20211204 IS - 1865-3774 (Electronic) IS - 0925-5710 (Linking) VI - 104 IP - 2 DP - 2016 Aug TI - Biomarkers for predicting clinical response to immunosuppressive therapy in aplastic anemia. PG - 153-8 LID - 10.1007/s12185-016-2009-z [doi] AB - The decision to select hematopoietic stem cell transplantation (HSCT) or immunosuppressive therapy (IST) as initial therapy in acquired aplastic anemia (AA) is currently based on patient age and the availability of a human leukocyte antigen (HLA)-matched donor. Although IST is a promising treatment option, the ability to predict its long-term outcomes remains poor due to refractoriness, relapses, and the risk of clonal evolution. Several predictive biomarkers for response to IST have been posited, including age, gender, pre-treatment blood cell counts, cytokines, gene mutations, paroxysmal nocturnal hemoglobinuria (PNH), and telomere length (TL). While previous studies have provided substantial biological insights into the utility of IST, the prognostic power of the reported biomarkers is currently insufficient to contribute to clinical decision making. Recently, a large retrospective analysis proposed the combination of minor PNH clones and TL as an efficient predictor of IST response. Identification of a reliable predictor would provide a useful tool for determining the most appropriate treatment choice for AA patients, including up-front HSCT from HLA-matched unrelated donor. The present review summarizes studies evaluating the utility of biomarkers in predicting the clinical response to IST of patients with AA, and provides a baseline for prospective studies aimed at validating previously reported biomarkers. FAU - Narita, Atsushi AU - Narita A AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, Aichi, 466-8650, Japan. FAU - Kojima, Seiji AU - Kojima S AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, Aichi, 466-8650, Japan. kojimas@med.nagoya-u.ac.jp. LA - eng PT - Journal Article PT - Review DEP - 20160418 PL - Japan TA - Int J Hematol JT - International journal of hematology JID - 9111627 RN - 0 (Biomarkers) SB - IM MH - Anemia, Aplastic/*therapy MH - Biomarkers/*analysis MH - Humans MH - Immunosuppression Therapy/*methods MH - Prognosis OTO - NOTNLM OT - Aplastic anemia OT - Immunosuppressive therapy OT - Paroxysmal nocturnal hemoglobinuria OT - Prognosis OT - Telomere length EDAT- 2016/04/20 06:00 MHDA- 2017/03/10 06:00 CRDT- 2016/04/20 06:00 PHST- 2016/03/09 00:00 [received] PHST- 2016/04/06 00:00 [accepted] PHST- 2016/04/06 00:00 [revised] PHST- 2016/04/20 06:00 [entrez] PHST- 2016/04/20 06:00 [pubmed] PHST- 2017/03/10 06:00 [medline] AID - 10.1007/s12185-016-2009-z [pii] AID - 10.1007/s12185-016-2009-z [doi] PST - ppublish SO - Int J Hematol. 2016 Aug;104(2):153-8. doi: 10.1007/s12185-016-2009-z. Epub 2016 Apr 18.