Long-acting growth hormone in the treatment of growth hormone deficiency in children: a systematic literature review and network meta-analysis.

The purpose of this study is to compare the relative efficacy and safety of long-acting growth hormone (LAGH) as a growth hormone replacement therapy in prepubertal children with growth hormone deficiency (GHD). We searched the PubMed, Embase, CNKI, and Wanfang databases from inception to July 2023 and identified eleven relevant studies. PEG-LAGH showed better effect on height velocity (mean difference [MD]: - 0.031, 95% credibility interval [CrI]: - 0.278, 0.215) than somatrogon (MD: 0.105, 95% CrI: - 0.419, 0.636), somapacitan (MD: 0.802, 95% CrI: - 0.451, 2.068) and lonapegsomatropin (MD: 1.335, 95% CrI: - 0.3, 2.989) when compared with daily growth hormone (DGH). Furthermore, in terms of height standard deviation score, PEG-LAGH demonstrated better improvement (MD: - 0.15, 95% CrI: - 1.1, 0.66) than somatrogon (MD: - 0.055, 95% CrI: - 1.3, 0.51) and somapacitan (MD: 0.22, 95% CrI: - 0.91, 1.3). PEG-LAGH (risk ratio [RR]: 1.00, 95% CrI: 0.82, 1.2) reduced the risk of adverse events compared with other LAGH (somatrogon, RR: 1.1, 95% CrI: 0.98, 1.2; somapacitan, RR: 1.1, 95% CrI: 0.96, 1.4; lonapegsomatropin, RR, 1.1, 95% CrI: 0.91, 1.3) and was comparable with DGH. This is the first study to indirectly compare the LAGH thorough a network meta-analysis and provide evidence of the optimal efficacy of various LAGH specifically PEG-LAGH and acceptable safety profile in prepubertal children with GHD.

Challenges and growth opportunities: the landscape of Chinese medical publication professionals in the global context: a questionnaire-based survey.

OBJECTIVE: The Chinese healthcare industry has immense potential for innovation and requires competent publication professionals for global reach. Changes in regulatory landscape of China necessitates pharmaceutical industries to collaborate with medical professionals who are capable of keeping up with both changes in regulations and innovative medicine. METHODS: We initiated an online questionnaire-based survey via WeChat from August 3 to August 14, 2019 which focused on experience level, challenges, career development, and training requirements of survey participants. RESULTS: A total of 53 medical publication professionals participated in this survey. Contrary to global trends, 77% of participants had <5 years of professional experience in publication, although only 36% of participants had <5 years of experience in healthcare industry. In all, 63% of respondents spent <50% of their time in publication-related activities, contributing to <10 manuscripts per year. Their expertise in manuscripts spanned clinical studies (68%), post-marketing studies (62%), and reviews (38%). Poor research design and data quality, limited medical writing skill, and inefficient communication with authors were major challenges for developing high-quality publications. Awareness of good publication practices guidelines was high (49%) and 15% of respondents were Certified Medical Publication Professionals. Strategic publication planning (72%), industry trends (66%), and best practices (55%) were some of training requirements identified to increase competencies among respondents. CONCLUSIONS: Although the Chinese medical publication professional industry is at a nascent stage, an attempt is being made to provide opportunities for its publication professionals to develop their competencies to match global standards.

Pharmaceutical industry-authored preprints: scientific and social media impact.

AIM: Non-peer-reviewed manuscripts posted as preprints can be cited in peer-reviewed articles, which has both merits and demerits. International Committee of Medical Journal Editors guidelines mandate authors to declare preprints at the time of manuscript submission. We evaluated the trends in pharma-authored research published as preprints and their scientific and social media impact by analyzing citation rates and altmetrics. RESEARCH DESIGN AND METHODS: We searched EuroPMC, PrePubMed bioRxiv and medRxiv for preprints submitted by authors affiliated with the top 50 pharmaceutical companies from inception until 15 June 2020. Data were extracted and analyzed from the search results. The number of citations for the preprint and peer-reviewed versions (if available) were compiled using the Publish or Perish software (version 1.7). Altmetric score was calculated using the "Altmetric it" online tool. Statistical significance was analyzed by Wilcoxon rank-sum test. RESULTS: A total of 498 preprints were identified across bioRxiv (83%), PeerJ (5%), F1000Research (6%), Nature Precedings (3%), Preprint.org (3%), Wellcome Open Research preprint (0.2%) and medRxiv (0.2%) servers. Roche, Sanofi and Novartis contributed 56% of the retrieved preprints. The median number of citations for the included preprints was 0 (IQR = 1, Min-Max = 0-45). The median number of citations for the published preprints and unpublished preprints was 0 for both (IQR = 1, Min-Max = 0-25 and IQR = 1, Min-Max = 0-45, respectively; p = .091). The median Altmetric score of the preprints was 4 (IQR = 10.5, Min-Max = 0-160). CONCLUSION: Pharma-authored research is being increasingly published as preprints and is also being cited in other peer-reviewed publications and discussed in social media.

Perspectives on the Early Quality of Evidence Guiding the Therapeutic Management of SARS-CoV-2: A Systematic Literature Review.

BACKGROUND: The severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) outbreak is a serious health concern. Repurposing of existing drugs indicated for other conditions seems to be the first choice for immediate therapeutic management. The quality of early evidence favoring the different treatment options needs to be apprised for informed decision-making. METHODS: In this systematic literature review, we apprised the quality of available evidence for different therapeutic options and also the basis for different treatment guidelines. To include all studies that are in different stages of publication, we also included studies from the preprint servers BioRxiv and MedRxiv and published studies from PubMed. RESULTS: We retrieved 5621 articles and included 22 studies for the systematic review. Based on our study, chloroquine/hydroxychloroquine, either alone or in combination with azithromycin, remdesivir, corticosteroids, convalescent sera, ritonavir/lopinavir, tocilizumab and arbidol were evaluated as therapeutic options. The data from different study designs reveal contradictory findings except for convalescent sera for which the evidence available is only from case series. Based on this early evidence, various national guidelines recommend remdesivir, convalescent sera, corticosteroids and hydroxychloroquine in different subsets of patients. CONCLUSION: Establishing consensus with respect to the end points to be assessed for respiratory viruses may enhance the quality of evidence in case of future pandemics. The systematic review highlighted the lacuna and methodologic deficiency in early clinical evidence and included an update on different therapeutic management guidelines. Further clinical evidence from the ongoing trials may lead to evolution of treatment guidelines with the addition of more therapeutic options.

Gefitinib as first line therapy in Malaysian patients with EGFR mutation-positive non-small-cell lung cancer: A single-center retrospective study.

The present retrospective, single-center study evaluated the objective response rate (ORR) and progression-free survival (PFS) of epidermal growth factor receptor (EGFR) mutation-positive Malaysian patients with advanced lung adenocarcinoma treated with gefitinib. During May 2008 to July 2013, 33 patients with Stage IV, EGFR mutation-positive non-small-cell lung cancer (NSCLC) were identified and received gefitinib (250 mg) as first line treatment. The primary and secondary end points were ORR, PFS and safety, respectively. A total of 18 (54.5%) and 2 (6.1%) patients achieved partial response (PR) and complete response (CR) to gefitinib therapy, respectively, yielding an ORR of 60.6% (95% CI, 42.1-77.1%). Patients with exon 20 or 21 mutations (n=6, 66.7%) tended to have better ORR compared with exon 19 (n=22, 59.1%). The median PFS was 8.9 months in Malaysian patients with EGFR mutation-positive NSCLC, treated with gefitinib. The majority of treatment-related toxicity was mild in nature. The most frequently reported adverse events included dry skin (39.4%), skin rash (27.2%), and dermatitis acneiform (15.2%). In conclusion, Malaysian patients with locally advanced and metastatic EGFR mutation-positive NSCLC responded favorably to gefitinib therapy in terms of ORR, median PFS, and tolerability, the results of which were consistent with those of the IPASS study conducted in an Asian population. Considering the efficacy and safety profile of gefitinib, it is a favorable option for the first-line treatment of Malaysian patients with EGFR mutation-positive NSCLC. However, future long-term studies in a larger population of Malaysian patients are required to support whether the prolonged PFS conferred by gefitinib will translate into prolonged overall survival.

Neurovascular preservation in female orthotopic radical cystectomy significantly improves sexual function.

Radical cystectomy has emerged as a standard treatment option for patients with muscle invasive bladder cancer. The impact of nerve-sparing cystectomy with urethra and vaginal sparing has not been quantified with validated questionnaires. We report our experience with nerve-sparing orthotopic radical cystectomy and the impact on postoperative sexual function.