scholarly journals Effectiveness and Safety of Sitagliptin in Patients with Beta-thalassaemia Major and Diabetes Mellitus: A Case Series

2016 ◽  
Vol 9 (1) ◽  
pp. e2017004 ◽  
Author(s):  
Shahrzad Zonoozi
Keyword(s):  
Diabetes Mellitus ◽  
Case Series ◽  
Close Monitoring ◽  
Thalassaemia Major ◽  
Efficacy And Safety ◽  
Antidiabetic Agent ◽  
Retrospective Case ◽  
Beta Thalassaemia ◽  
Equivalent Reduction

Sitagliptin, a modern antidiabetic agent which is weight neutral and associated with low rate of hypoglycaemias, is being increasingly used in type 2 diabetes mellitus (DM). However there is paucity of data about its efficacy and safety in beta-thalassaemia major (β-TM).This retrospective case series of five patients (mean age of 45 years) is the first study evaluating the use of sitagliptin in patients with β-TM and DM.Four patients responded well to sitagliptin, as evidenced by decrease in fructosamine by 77 and 96µmol/L (equivalent reduction in HbA1c of 1.5% and 1.9%) observed in two patients and reduction in the frequency of hypoglycaemia without worsening glycaemic control in two others. One patient did not respond to sitagliptin. No patients reported significant side effects.This study provides evidence that sitagliptin may be considered, with caution, for use in patients with β-TM and DM, under the close monitoring of a Diabetologist.

Long-Term Efficacy and Safety of Ertugliflozin in Patients (Pts) with Type 2 Diabetes Mellitus (T2DM) Inadequately Controlled with Metformin (MET) Monotherapy—104-Week VERTIS MET Trial

Diabetes ◽  
2018 ◽  
Vol 67 (Supplement 1) ◽  
pp. 1129-P
Author(s):  
SILVINA GALLO ◽  
BERNARD CHARBONNEL ◽  
ALLISON GOLDMAN ◽  
HARRY SHI ◽  
SUSAN HUYCK ◽  
...  
Keyword(s):  
Diabetes Mellitus ◽  
Type 2 Diabetes ◽  
Type 2 Diabetes Mellitus ◽  
Efficacy And Safety ◽  
Term Efficacy

Efficacy and Safety of Continuing Sitagliptin when Initiating Insulin Therapy in Subjects with Type 2 Diabetes Mellitus

Diabetes ◽  
2018 ◽  
Vol 67 (Supplement 1) ◽  
pp. 112-LB ◽  
Author(s):  
RONAN ROUSSEL ◽  
SANTIAGO DURAN-GARCIA ◽  
YILONG ZHANG ◽  
SUNERI SHAH ◽  
CAROLYN DARMIENTO ◽  
...  
Keyword(s):  
Diabetes Mellitus ◽  
Type 2 Diabetes ◽  
Type 2 Diabetes Mellitus ◽  
Insulin Therapy ◽  
Efficacy And Safety

Efficacy and Safety of Evolocumab in Patients with Type 2 Diabetes Mellitus and Hypercholesterolemia or Mixed Dyslipidemia

Diabetes ◽  
2018 ◽  
Vol 67 (Supplement 1) ◽  
pp. 128-OR ◽  
Author(s):  
ROBERT S. ROSENSON ◽  
MARTHA L. DAVIGLUS ◽  
PETER REAVEN ◽  
PAOLO POZZILLI ◽  
HAROLD BAYS ◽  
...  
Keyword(s):  
Diabetes Mellitus ◽  
Type 2 Diabetes ◽  
Type 2 Diabetes Mellitus ◽  
Efficacy And Safety ◽  
Mixed Dyslipidemia

scholarly journals Cerliponase Alfa for the Treatment of Atypical Phenotypes of CLN2 Disease: A Retrospective Case Series

2020 ◽  
pp. 088307382097799
Author(s):  
Eva Wibbeler ◽  
Raymond Wang ◽  
Emily de los Reyes ◽  
Nicola Specchio ◽  
Paul Gissen ◽  
...  
Keyword(s):  
Treatment Duration ◽  
Chart Review ◽  
Rating Scale ◽  
Neuronal Ceroid Lipofuscinosis ◽  
Case Series ◽  
Language Function ◽  
Retrospective Case ◽  
Function Loss ◽  
Clinical Rating Scale

Background: The classic phenotype of CLN2 disease (neuronal ceroid lipofuscinosis type 2) typically manifests between ages 2 and 4 years with a predictable clinical course marked by epilepsy, language developmental delay, and rapid psychomotor decline. Atypical phenotypes exhibit variable time of onset, symptomatology, and/or progression. Intracerebroventricular-administered cerliponase alfa (rhTPP1 enzyme) has been shown to stabilize motor and language function loss in patients with classic CLN2 disease, but its impact on individuals with atypical phenotypes has not been described. Methods: A chart review was conducted of 14 patients (8 male, 6 female) with atypical CLN2 phenotypes who received cerliponase alfa. Pre- and posttreatment CLN2 Clinical Rating Scale Motor and Language (ML) domain scores were compared. Results: Median age at first presenting symptom was 5.9 years. First reported symptoms were language abnormalities (6 [43%] patients), seizures (4 [29%]), ataxia/language abnormalities (3 [21%]), and ataxia alone (1 [7%]). Median age at diagnosis was 10.8 years. ML score declined before treatment in 13 (93%) patients. Median age at treatment initiation was 11.7 years; treatment duration ranged from 11 to 58 months. From treatment start, ML score remained stable in 11 patients (treatment duration 11-43 months), improved 1 point in 1 patient after 13 months, and declined 1 point in 2 patients after 15 and 58 months, respectively. There were 13 device-related infections in 8 patients (57%) and 10 hypersensitivity reactions in 6 (43%). Conclusions: Cerliponase alfa is well tolerated and has the potential to stabilize motor and language function in patients with atypical phenotypes of CLN2 disease.

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