Biomarkers for predicting clinical response to immunosuppressive therapy in aplastic anemia

Narita, Atsushi; Kojima, Seiji

doi:10.1007/s12185-016-2009-z

Cited by 14 publications

(12 citation statements)

References 43 publications

(53 reference statements)

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“…In SAA, age, sex, and pre-treatment PB counts are established as prognostically valuable markers for response to IST. 50 In the current study, we observed positive correlation of some exosomal miRNAs with WBC and Plt counts, and LDH levels at diagnosis in SAA, but not with pre-treatment PB counts. These results suggest that their expression was not affected by transfusion history or disease severity, and that miRNAs could be used as independent diagnostic or prognostic markers.…”

Section: Discussionsupporting

confidence: 52%

Circulating exosomal microRNAs in acquired aplastic anemia and myelodysplastic syndromes

et al. 2018

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Exosomal microRNAs modulate cancer cell metabolism and the immune response. Specific exosomal microRNAs have been reported to be reliable biomarkers of several solid and hematologic malignancies. We examined the possible diagnostic and prognostic values of exosomal microRNAs in two human bone marrow failure diseases: aplastic anemia and myelodysplastic syndromes. After screening 372 microRNAs in a discovery set (n=42) of plasma exosome samples, we constructed a customized PCR plate, including 42 microRNAs, for validation in a larger cohort (n=99). We identified 25 differentially expressed exosomal microRNAs uniquely or frequently present in aplastic anemia and/or myelodysplastic syndromes. These microRNAs could be related to intracellular functions, such as metabolism, cell survival, and proliferation. Clinical parameters and progression-free survival were correlated to microRNA expression levels in aplastic anemia and myelodysplastic syndrome patients before and after six months of immunosuppressive therapy. One microRNA, mir-126-5p, was negatively correlated with a response to therapy in aplastic anemia: patients with higher relative expression of miR-126-5p at diagnosis had the shortest progression-free survival compared to those with lower or normal levels. Our findings suggest utility of exosomal microRNAs in the differential diagnosis of bone marrow failure syndromes. (Registered at clinicaltrials.gov identifiers: 00260689, 00604201, 00378534, 01623167, 00001620, 00001397, 00217594).

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Section: Discussionsupporting

confidence: 52%

Circulating exosomal microRNAs in acquired aplastic anemia and myelodysplastic syndromes

et al. 2018

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“…Flow cytometry is more sensitive than PIGA gene sequencing for detecting the presence of small clones . The clinical significance of PNH clones in aAA is uncertain, but several studies have reported that patients who harbour a PNH clone have a favourable response to IST . In some cases, small PNH clones can expand to cause clinically overt PNH.…”

Section: Pathogenesismentioning

confidence: 99%

Revisiting acquired aplastic anaemia: current concepts in diagnosis and management

Clucas

Fox

Wood

et al. 2019

Internal Medicine Journal

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Acquired aplastic anaemia is a rare, serious, immunologically mediated bone marrow failure syndrome, characterised by marrow hypoplasia of varying severity and significant pancytopenia. Careful attention and investigation, including molecular testing, is required to confirm the diagnosis and exclude other mimicking conditions, such as inherited bone marrow failure syndromes. In a proportion of patients, the disease evolves to myelodysplasia or acute myeloid leukaemia and in some there is an association with paroxysmal nocturnal haemoglobinuria. The disease has a major impact on patient quality of life. Haemopoietic stem/progenitor cell transplantation for eligible patients with an available donor is the only current curative therapy. Other patients may receive immunosuppression, most commonly with anti-thymocyte globulin and cyclosporin. An initial response to immunosuppression is often encouraging, but relapse is common. Supportive care, including management of transfusion requirements and infections, is central to management. Promising new diagnostic tools and emerging therapies will likely transform approaches to this important, chronic and lifethreatening condition.

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“…Shorter duration of AA, younger age at diagnosis, a higher absolute neutrophil count, a higher absolute lymphocyte count, a higher baseline platelet count, higher CsA levels during the first 2 weeks of commencement of IST, longer telomere length, positive minor PNH clone, lower thrombopoietin (TPO) levels, and increased IFN-gamma expression in the bone marrow indicate better response to IST. 11 Telomere shortens with age and is a marker of an aging process. Significant abnormal shortening of telomeres of bone marrow nucleated cells have been seen in elderly AA.…”

Section: Discussionmentioning

confidence: 99%

“…Several other biomarkers may contribute to responsiveness of immunosuppressive therapy (IST) in elderly AA. Shorter duration of AA, younger age at diagnosis, a higher absolute neutrophil count, a higher absolute lymphocyte count, a higher baseline platelet count, higher CsA levels during the first 2 weeks of commencement of IST, longer telomere length, positive minor PNH clone, lower thrombopoietin (TPO) levels, and increased IFN‐gamma expression in the bone marrow indicate better response to IST 11 …”

Section: Discussionmentioning

confidence: 99%

Very severe aplastic anemia in an 80‐year‐old man

Kasinathan

Lee

Sathar

2021

Clinical Case Reports

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Biomarkers for predicting clinical response to immunosuppressive therapy in aplastic anemia

Cited by 14 publications

References 43 publications

Circulating exosomal microRNAs in acquired aplastic anemia and myelodysplastic syndromes

Circulating exosomal microRNAs in acquired aplastic anemia and myelodysplastic syndromes

Revisiting acquired aplastic anaemia: current concepts in diagnosis and management

Very severe aplastic anemia in an 80‐year‐old man

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