Treatment patterns and outcomes in children with infantile hemangiomas: A retrospective observational analysis.
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ABSTRACT: Objective
This study aimed to make use of real-world medical records to explore the clinical characteristics, treatments, and outcomes of infantile hemangiomas in southeastern China.Methods
This study applied a retrospective observational method using real-world data derived from the electronic medical records of the Foshan Women and Children Hospital, southeastern China dated between June 2014 and June 2019.Results
A total of 2427 patients with infantile hemangiomas were recruited in this study, including 942 (38.8%) males and 1485 (61.2%) females. Among the participants, 620 (25.5%) were high-risk infantile hemangioma, 449 (18.5%) were medium risk, and 1358 (56.0%) were low risk. A total of 14 treatment patterns in clinical practice were identified. The top 3 treatment patterns in each group of risk levels were the same: laser therapy, a combination of laser therapy and topical timolol maleate, and topical timolol maleate. The outcomes of the top 3 treatment patterns were significantly (P < 0.05) different in each risk group.Conclusion
Among the top 3 treatment patterns, laser therapy or a combination of laser therapy and topical timolol maleate were more likely to have an "Excellent" outcome.
SUBMITTER: Cai Y
PROVIDER: S-EPMC8685728 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:Compound infantile hemangiomas (IHs) are problematic and usually require intervention. This retrospective study aimed to introduce a combined therapy of oral propranolol and topical timolol, and evaluate its efficacy and safety. Eighty-nine infants with compound IHs were treated with oral propranolol 2 mg/kg/day divided 2 times per day and timolol maleate 0.5% gel 3 times per day, for at least 3 months. Two observers evaluated the hemangioma independently at 0, 1, 3, 6, 9 months after the initiation of treatment. Changes in the hemangioma score values were evaluated using paired t test. Rebound growth and adverse effects were recorded. After treatment was completed, this combined therapy achieved clinical response in 100% of the patients (89/89). Significant positive effects were demonstrated at 1, 3, 6 months (p < 0.001), but not obvious after 6 months (p = 0.06). The response of IHs to the therapy was depending on the age at initial treatment. The average treatment duration was 6.48 (5.77-7.19) months. One patient (1.1%) relapsed after cessation of 6-month treatment, and 7 children (7.8%) developed side effects. Our study suggested that oral propranolol combined with topical timolol treatment is very effective and well-tolerated for compound IHs, which can be used as a first line treatment.
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Project description:BackgroundInfantile Hemangiomas (IHs) are common benign vascular tumors of infancy that may have serious consequences. The research on diagnostic markers for IHs is scarce.MethodsThe "limma" R package was applied to identify differentially expressed genes (DEGs) in developing IHs. Plugin ClueGO in Cytoscape software performed functional enrichment of DEGs. The Search Tool for Retrieving Interacting Genes (STRING) database was utilized to construct the PPI network. The least absolute shrinkage and selection operator (LASSO) regression model and support vector machine recursive feature elimination (SVM-RFE) analysis were used to identify diagnostic genes for IHs. The receiver operating characteristic (ROC) curve evaluated diagnostic genes' discriminatory ability. Single-gene based on Gene Ontology (GO) and Kyoto Encyclopedia of Genes and Genomes (KEGG) was conducted by Gene Set Enrichment Analysis (GSEA). The chemicals related to the diagnostic genes were excavated by the Comparative Toxicogenomics Database (CTD). Finally, the online website Network Analyst was used to predict the transcription factors targeting the diagnostic genes.ResultsA total of 205 DEGs were singled out from IHs samples of 6-, 12-, and 24-month-old infants. These genes principally participated in vasculogenesis and development-related, endothelial cell-related biological processes. Then we mined 127 interacting proteins and created a network with 127 nodes and 251 edges. Furthermore, LASSO and SVM-RRF algorithms identified five diagnostic genes, namely, TMEM2, GUCY1A2, ISL1, WARS, and STEAP4. ROC curve analysis results indicated that the diagnostic genes had a powerful ability to distinguish IHs samples from normal samples. Next, the results of GSEA for a single gene illustrated that all five diagnostic genes inhibited the "valine, leucine, and isoleucine degradation" pathway in the development of IHs. WARS, TMEM2, and STEAP4 activated the "blood vessel development" and "vasculature development" in IHs. Subsequently, inhibitors targeting TMEM2, GUCY1A2, ISL1, and STEAP4 were mined. Finally, 14 transcription factors regulating GUCY1A2, 14 transcription factors regulating STEAP4, and 26 transcription factors regulating ISL1 were predicted.ConclusionThis study identified five diagnostic markers for IHs and further explored the mechanisms and targeting drugs, providing a basis for diagnosing and treating IHs.
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Project description:Infantile hemangiomas are the most common type of benigh tumors in early childhood, and propanolol is a standard treatment with yet unknown mechanism of action. In this study, we identify putative biomarkers for infantile hemangioma stages (proliferative and involution) in the context of propanolol treatment.
2019-03-01 | GSE127487 | GEO
Project description:BackgroundPancreatic cancer represents the third leading cause of US cancer deaths, with median survival <1 year. The goal of this study was to describe systemic treatments, healthcare utilization and costs, and overall survival among patients with unresectable/metastatic disease.MethodsThis study used healthcare claims for commercial and Medicare Advantage enrollees diagnosed with pancreatic adenocarcinoma (at index date) during January 01 2010 to 31 May 2017. Included patients were aged ≥18 years, with continuous 6-month preindex enrollment. Patients were excluded by resectable disease, another primary cancer, or pregnancy. Cohorts were based on first-line (LOT1) chemotherapy regimen.ResultsOverall, 12 978 patients (mean age 70 years, 51% male) were included, among which 5610 (43%) received chemotherapy. Of those, 23% received gemcitabine monotherapy, 22% gemcitabine-nab paclitaxel, 22% FOLFIRINOX, 3% FOLFOX, and 29% received other regimens. Mean LOT1 duration was 112 days; 60% did not undergo subsequent lines of therapy. Moreover, 50% of patients had an emergency room visit and 45% were hospitalized during LOT1. Among treated and untreated patients, mean total 6-month costs were $52 101. We found that patients receiving FOLFIRINOX had the highest costs, whereas those who received gemcitabine monotherapy had the lowest. Median overall survival (mOS) was 335 days with any first-line treatment. FOLFIRINOX-treated patients had the highest mOS (492 days), whereas gemcitabine monotherapy-treated patients had the lowest (223 days).ConclusionsA large proportion (57%) of patients with unresectable/metastatic pancreatic cancer did not receive chemotherapy. Healthcare costs were higher for fluorouracil-based regimens, while lower for gemcitabine-based regimens. Survival rates were within expectations for advanced pancreatic cancer.
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Project description:The COVID-19 pandemic has caused significant shifts in patient care including a steep decline in ambulatory visits and a marked increase in the use of telemedicine. Infantile hemangiomas (IH) can require urgent evaluation and risk stratification to determine which infants need treatment and which can be managed with continued observation. For those requiring treatment, prompt initiation decreases morbidity and improves long-term outcomes. The Hemangioma Investigator Group has created consensus recommendations for management of IH via telemedicine. FDA/EMA-approved monitoring guidelines, clinical practice guidelines, and relevant, up-to-date publications regarding initiation and monitoring of beta-blocker therapy were used to inform the recommendations. Clinical decision-making guidelines about when telehealth is an appropriate alternative to in-office visits, including medication initiation, dosage changes, and ongoing evaluation, are included. The importance of communication with caregivers in the context of telemedicine is discussed, and online resources for both hemangioma education and propranolol therapy are provided.
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Project description:BackgroundEpidemiological studies evaluating treatments for infantile hemangiomas have produced inconsistent results. A meta-analysis of published data was conducted to investigate the effectiveness and safety of oral propranolol versus other treatments for infantile hemangiomas.MethodsA meta-analysis was conducted based on literature (published from 1960 to December 1, 2014) found on the PubMed, EMBASE, and OVID search engines. Pooled odds ratios (ORs) and 95% confidence intervals (CIs) were estimated for the outcome measures. Heterogeneity, publication bias and subgroup analysis were performed.ResultsA total of 61 studies involving 5,130 participants met the inclusion criteria. Propranolol was found to be a more effective modality in treating IHs (ORs = 0.92; 95%CI, 0.89-0.95) and had fewer complications compared to the other treatments including systemic steroids (ORs = 0.68; 95% CI, 0.59-0.76); laser ablation (ORs = 0.55; 95% CI, 0.43-0.67); other beta-adrenergic blockers (ORs = 0.56; 95% CI, 0.50-0.61) and surgery (ORs = 0.55; 95% CI, 0.28-0.81). A subgroup analysis of propranolol showed that a dose of 2 mg/kg/day or more yielded better outcomes (ORs = 0.92; 95% CI, 0.88-0.95; ORs = 0.95; 95% CI, 0.89-1.00), and IHs that had not been previously treated had better responses to propranolol treatment (ORs = 0.95; 95% CI, 0.91-0.98).ConclusionsThe meta-analysis demonstrated that propranolol was more effective and safer than other therapies in treating IHs. It provides strong evidence for supporting the use of propranolol as a first-line therapy for IHs.
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