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Cseh A.M.,Albert Ludwigs University of Freiburg | Niemeyer C.M.,Albert Ludwigs University of Freiburg | Yoshimi A.,Albert Ludwigs University of Freiburg | Catala A.,Hospital Sant Joan de Deu | And 15 more authors.
British Journal of Haematology | Year: 2016

Low-dose azacitidine is efficient and safe in the therapy of malignant myeloid disorders in adults but data in children are lacking. We present a retrospective analysis of 24 children and young adults with myelodysplastic syndrome (MDS) who received azacitidine at the time of first diagnosis or relapse after allotransplant (2 children were treated with azacitidine both initially and for relapse). Diagnoses were refractory cytopenia of childhood (N = 4), advanced primary MDS (N = 9) and secondary MDS (N = 11). The median duration of treatment was four cycles. Azacitidine was well tolerated, but cytopenias led to dose reduction in five cases. Treatment was discontinued in one child because of impaired renal function. Sixteen MDS patients were treated with azacitidine at first diagnosis. One complete clinical remission was observed and one child showed complete marrow remission; six children experienced stable disease with haematological improvement. Ten children received azacitidine for relapsed MDS after transplant: of these, seven experienced stable disease for 2-30 cycles (median 3), including one patient with haematological improvement for seven cycles. In summary, azacitidine is effective in some children with MDS and appears to be a non-toxic option in palliative situations to prolong survival. © 2016 John Wiley & Sons Ltd.

Bayazit Y.A.,Istanbul University | Kosaner J.,Meders Hearing and Speech Center | Cinar B.C.,Meders Hearing and Speech Center | Atac A.,Istanbul University | And 7 more authors.
Annals of Otology, Rhinology and Laryngology | Year: 2014

Objective: The objective was to provide information about methods used and preliminary outcomes for pediatric ABI (auditory brainstem implant). Study Design: An analysis of outcome was performed in children who received an ABI. Methods: Twelve children received a MED-EL ABI system. Progress in audition and language was monitored through parental reports, questionnaires, profiles, and closed-set tests. Results: The median number of active electrodes was 9 of 12. Seven of 12 users consistently respond to sound, and 5 of 12 do not. Highest performers can recognize words in small sets and have begun to use some words. Conclusion: Auditory brainstem implants appear to be beneficial for some pediatric patients who cannot benefit from traditional cochlear implant surgery. Benefits in the short term can be recognition of environmental sounds, recognition of some words and very commonly used phrases, and the beginning use of words. Although some of our ABI users demonstrate no response to sound, they do want to wear their sound processors all waking hours. The cause of lack of response may be related to the second intervention, which might have led to displacement of the electrode array, or presence of additional handicaps or syndromes. However, the results are less than optimal. The relatively short postoperative follow-up duration is a considered weakness of this study. © The Author(s) 2014.

Gasparini P.,Ohio State University | Cascione L.,Ohio State University | Cascione L.,Ior Instituteofoncologyresearch | Fassan M.,Ohio State University | And 9 more authors.
Oncotarget | Year: 2014

Triple Negative Breast Cancers (TNBC) is a heterogeneous disease at the molecular and clinical level with poor outcome. Molecular subclassification of TNBCs is essential for optimal use of current therapies and for development of new drugs. microRNAs (miRNA) are widely recognized as key players in cancer progression and drug resistance; investigation of their involvement in a TNBC cohort may reveal biomarkers for diagnosis and prognosis of TNBC. Here we stratified a large TNBC cohort into Core Basal (CB, EGFR and/or CK5, 6 positive) and five negative (5NP) if all markers are negative. We determined the complete miRNA expression profile and found a subset of miRNAs specifically deregulated in the two subclasses. We identified a 4-miRNA signature given by miR-155, miR-493, miR-30e and miR- 27a expression levels, that allowed subdivision of TNBCs not only into CB and 5NP subgroups (sensitivity 0.75 and specificity 0.56; AUC=0.74) but also into high risk and low risk groups. We tested the diagnostic and prognostic performances of both the 5 IHC marker panel and the 4-miRNA expression signatures, which clearly identify worse outcome patients in the treated and untreated subcohorts. Both signatures have diagnostic and prognostic value, predicting outcomes of patient treatment with the two most commonly used chemotherapy regimens in TNBC: anthracycline or anthracycline plus taxanes. Further investigation of the patients' overall survival treated with these regimens show that regardless of IHC group subdivision, taxanes addition did not benefit patients, possibly due to miRNA driven taxanes resistance. TNBC subclassification based on the 5 IHC markers and on the miR-155, miR-493, miR-30e, miR-27a expression levels are powerful diagnostic tools. Treatment choice and new drug development should consider this new subtyping and miRNA expression signature in planning low toxicity, maximum efficacy therapies.

Yalcin E.,Research Hospital of the Ministry of Health | Unlu E.,Diskapi Yildirim Beyazit Training and Research Hospital | Akyuz M.,Research Hospital of the Ministry of Health | Karaahmet O.Z.,Diskapi Yildirim Beyazit Training and Research Hospital
Journal of Hand Surgery: European Volume | Year: 2014

There is still no consensus on a normal value for the cross-sectional area of the ulnar nerve at the elbow. Such data would be valuable for the ultrasound diagnosis of ulnar neuropathy. Comparison of the symptomatic and contralateral asymptomatic sides has been recommended. The aim of this study was to investigate whether or not the asymptomatic ulnar nerve could be a reference value. High-resolution ultrasonic measurements of the cross-sectional areas of the ulnar nerves at the elbow were compared with 38 contralateral asymptomatic elbows of patients with unilateral ulnar neuropathy and 38 healthy controls. There were significant differences in the cross-sectional areas at all levels between the healthy control subjects and asymptomatic side of the ulnar neuropathy patients (p < 0.05). The findings of this study suggest that there is a limitation in using the asymptomatic side for obtaining reference values in ultrasonographic studies. Each ultrasonographic laboratory should determine its own reference values. © The Author(s) 2013.

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