| 1. |
- Hageman, Isabel C., et al.
(author)
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A Quality Assessment of the ARM-Net Registry Design and Data Collection
- 2023
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In: Journal of Pediatric Surgery. - : Elsevier BV. - 0022-3468. ; 58:10, s. 1921-1928
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Journal article (peer-reviewed)abstract
- Background: Registries are important in rare disease research. The Anorectal Malformation Network (ARM-Net) registry is a well-established European patient registry collecting demographic, clinical, and functional outcome data. We assessed the quality of this registry through review of the structure, data elements, collected data, and user experience. Material and methods: Design and data elements were assessed for completeness, consistency, usefulness, accuracy, validity, and comparability. An intra- and inter-user variability study was conducted through monitoring and re-registration of patients. User experience was assessed via a questionnaire on registration, design of registry, and satisfaction. Results: We evaluated 119 data elements, of which 107 were utilized and comprised 42 string and 65 numeric elements. A minority (37.0%) of the 2278 included records had complete data, though this improved to 83.5% when follow-up elements were excluded. Intra-observer variability demonstrated 11.7% incongruence, while inter-observer variability was 14.7%. Users were predominantly pediatric surgeons and typically registered patients within 11–30 min. Users did not experience any significant difficulties with data entry and were generally satisfied with the registry, but preferred more longitudinal data and patient-reported outcomes. Conclusions: The ARM-Net registry presents one of the largest ARM cohorts. Although its collected data are valuable, they are susceptible to error and user variability. Continuous evaluations are required to maintain relevant and high-quality data and to achieve long-term sustainability. With the recommendations resulting from this study, we call for rare disease patient registries to take example and aim to continuously improve their data quality to enhance the small, but impactful, field of rare disease research. Level of Evidence: V.
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| 2. |
- Inci, Ilyas, et al.
(author)
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Gelatin-Hydroxyapatite Cryogels with Bone Morphogenetic Protein-2 and Transforming Growth Factor Beta-1 for Calvarial Defects
- 2014
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In: Journal of Biomaterials and Tissue Engineering. - : American Scientific Publishers. - 2157-9091 .- 2157-9083. ; 4:8, s. 624-631
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Journal article (peer-reviewed)abstract
- Over recent years, several tissue engineering techniques are used for bone regeneration. In this study gelatin-hydroxyapatite (Gelatin-HA) cryogel scaffolds were used with/without bone morphogenetic protein-2 (BMP-2) and transforming growth factor beta-1 (TGF-beta 1) to heal critical sized bone defects. Release of growth factors from the cryogels were performed up to 28 days in vitro. Moreover, in vivo study were performed by 6 groups and results were evaluated by micro computerized tomography (mu-CT) and histology on 2 months and 4 months after surgery. Gene expression studies were also performed on 2 weeks and 4 weeks after surgery. Collagen type I (Col I), alkaline phosphatase (ALP) and osteocalcin (OSC) gene expression levels were analyzed. According to histology and mu-CT results, there was a significant bone healing in the BMP-2 containing gelatin-HA cryogel group (Group I) as compared to other groups except autograft applied group (Group IV). Moreover, significant increases were observed for Col I, ALP and OSC in Group I and Group IV. However there was no significant increase in the group of BMP-2 and TGF-beta 1 applied together (Group II). The results reveal that combination of gelatin-HA cryogels with BMP-2 is a promising candidate for healing in critical sized craniofacial bone defects.
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