enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3

alexandrossfakianakis shared this article with you from Inoreader Comprehensive and long‐term outcomes of enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3 via Pediatric Blood & Cancer Abstract Background Gaucher disease (GD) is a lysosomal storage disorder, characterized by hepatosplenomegaly, pancytopenia, bone diseases, with or without neurological symptoms. Plasma glucosylsphingosine (lyso-Gb1), a highly sensitive and specific biomarker for GD, has been used for diagnosis and monitoring the response to treatment. Enzyme replacement therapy (ERT) is an effective treatment for the non-neurologic symptoms of GD. Neuronopathic GD (type 2 and 3) accounts for 60%–70% of the Asian affected population. Methods We explored combination therapy of ERT followed by hematopoietic stem cell transplantation (HSCT) and its long-term outcomes in patients with GD type 3 (GD3). Results Four patients with GD3 and one with GD type 1 (GD1) underwent HSCT. The types of donor were one matched-related, one matched-unrelated, and three haploidentical. The age at disease onset was 6–18 months and the age at HSCT was 3.8–15 years in the patients with GD3. The latest age at follow-up was 8–22 years, with a post-HSCT duration of 3–14 years. All patients had successful HSCT. Chronic graft-versus-host disease occurred in one patient. The enzyme activities were normalized at 2 weeks post HSCT. Lyso-Gb1 concentrations became lower than the pathological value. All of the patients are still alive and physically independent. Most of them (4/5) returned to school. None of the patients with GD3 had seizures or additional neurological symptoms after HSCT, but showed varying degrees of cognitive impairment. Conclusions ERT followed by HSCT could be considered as an alternative treatment for patients with GD3 who have a high risk of fatal neurological progression. View on Web

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high‐viscosity glass ionomer restorative systems in small class II restorations

alexandrossfakianakis shared this article with you from Inoreader Five‐year randomized clinical trial to evaluate the clinical performance of high‐viscosity glass ionomer restorative systems in small class II restorations via Journal of Esthetic and Restorative Dentistry Abstract Objective Evaluate and compare the 5-year clinical performance of three high-viscosity glass ionomer restorative materials in small class II restorations. Materials and Methods Forty patients, each with four class II restorations, were enrolled in this trial. A total of 160 restorations were placed, 25% for each material, as follows: three high-viscosity conventional glass ionomer restorative systems (Ketac Universal Aplicap, EQUIA Forte and Riva Self Cure HV) and a microhybrid resin composite system (Filtek Z250). Clinical evaluation was performed at baseline and after 1, 3, and 5 years by two independent examiners using FDI criteria. Epoxy resin replicas were observed under scanning electron microscope (SEM) to examine surface characteristics. Data were analyzed with Kruskal-Wallis, Mann–Whitney U, Friedman, and Wilcoxon signed-rank tests (p < 0.05). Results The success rates were 100% for resin composite, 97.4% for Ketac Universal, and 94.9% for both EQUIA Forte and Riva HV restorations. Statistically significant differences were observed between all groups in terms of surface luster and color match criteria (p < 0.05). Statistically significant changes were found over time for all criteria except for fracture of material, postoperative hypersensitivity, recurrence of caries, tooth integrity, periodontal response, adjacent mucosa, and oral health criteria (p > 0.05). SEM evaluations were in accordance with the clinical findings. Conclusions Although drawbacks in surface luster and color match appeared over the 5-year evaluation period, the three high-viscosity glass ionomer restorative materials provided successful clinical performance in small to medium sized class II cavities compared to microhybrid resin composite. Clinical Significance Glass ionomer restorations exhibited clinical performance similar to that of microhybrid resin composite restorations in small class II cavities subsequent to 5-year evaluation. View on Web

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