Gene therapy for haemophilia.

BACKGROUND: Haemophilia is a genetic disorder characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft tissues. Current methods of treatment are expensive, challenging and involve regular administration of clotting factors. Gene therapy for haemophilia is a curative treatment modality currently under investigation. This is an update of a published Cochrane Review. OBJECTIVES: To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis & Genetic Disorders Group's Coagulopathies Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. We also searched the reference lists of relevant articles and reviews. Date of last search: 17 April 2020. SELECTION CRITERIA: Eligible trials include randomised or quasi-randomised clinical trials, including controlled clinical trials comparing gene therapy (with or without standard treatment) with standard treatment (factor replacement) or other 'curative' treatment such as stem cell transplantation for individuals with haemophilia A or B of all ages who do not have inhibitors to factor VIII or IX. DATA COLLECTION AND ANALYSIS: No trials of gene therapy for haemophilia matching the inclusion criteria were identified. MAIN RESULTS: No trials of gene therapy for haemophilia matching the inclusion criteria were identified. AUTHORS' CONCLUSIONS: No randomised or quasi-randomised clinical trials of gene therapy for haemophilia were identified. Thus, we are unable to determine the safety and efficacy of gene therapy for haemophilia. Gene therapy for haemophilia is still in clinical investigation and there is a need for well-designed clinical trials to assess the long-term feasibility, success and risks of gene therapy for people with haemophilia.

Parenteral Versus Oral Iron for Treatment of Iron Deficiency Anaemia During Pregnancy and post-partum: A Systematic Review.

INTRODUCTION: The burden of iron deficiency anaemia during pregnancy and post-partum continues to remain high especially in India. Challenges to treatment include gastrointestinal side effects and non compliance to oral iron therapy. Newer parenteral formulations need to be explored as alternatives. METHODS: Meta-analysis of randomized controlled trials published between years 2011 and 2018 comparing anaemic pregnant and post-partum women treated with intravenous iron sucrose versus oral iron was performed. The primary outcomes were mean maternal haemoglobin, serum ferritin and haematocrit at the end of 1st, 2nd, 4th and 6th weeks and comparison of adverse effects. RESULTS: Eighteen studies including 1633 antenatal women were randomly assigned to intravenous iron sucrose (n = 821) or oral iron [ferrous sulphate, ferrous ascorbate or fumarate] group (n = 812) in ten trials . Another eight studies compared iron sucrose infusion with oral iron in 713 post-partum women who were randomly assigned to intravenous iron sucrose group (n = 351) or oral iron group (n = 362). Cumulative analysis of all the time points indicates that the estimated mean values of Hb in the intravenous iron sucrose and oral iron groups were 10.11 g/dl and 9.33 g/dl, respectively, in antenatal group, while it was 10.57 g/dl and 9.74 g/dl in post-partum. The estimated mean ferritin level from first week to six weeks was 63.1 µg/l and 28.6 µg/l, respectively, in intravenous and oral iron groups. Cumulative estimate of haematocrit in the intravenous sucrose and oral iron over 6 weeks showed that the mean values in the respective groups were 30.5% and 29.5% in antenatal and 33.8% and 31.6%, respectively, in post-partum groups. Sensitivity analysis confirmed the reliability and consistency of the results. Oral iron was associated with significant gastrointestinal side effects. There was no significant difference in birthweight between the groups. CONCLUSION: This meta-analysis demonstrates that intravenous iron sucrose is more effective than oral iron therapy for pregnant and post-partum women with iron deficiency anaemia. It is an effective and safe alternative to address the problem of iron deficiency especially in those who require rapid replacement of iron stores though medical personnel for intravenous administration of drug is required.Trial registration CRD42015024343.

Gene therapy for haemophilia.

BACKGROUND: Haemophilia is a genetic disorder characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft tissues. Current methods of treatment are expensive, challenging and involve regular administration of clotting factors. Gene therapy has recently been prompted as a curative treatment modality. This is an update of a published Cochrane Review. OBJECTIVES: To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis & Genetic Disorders Group's Coagulopathies Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. We also searched the reference lists of relevant articles and reviews.Date of last search: 18 August 2016. SELECTION CRITERIA: Eligible trials include randomised or quasi-randomised clinical trials, including controlled clinical trials comparing gene therapy (with or without standard treatment) with standard treatment (factor replacement) or other 'curative' treatment such as stem cell transplantation for individuals with haemophilia A or B of all ages who do not have inhibitors to factor VIII or IX. DATA COLLECTION AND ANALYSIS: No trials of gene therapy for haemophilia were found. MAIN RESULTS: No trials of gene therapy for haemophilia were identified. AUTHORS' CONCLUSIONS: No randomised or quasi-randomised clinical trials of gene therapy for haemophilia were identified. Thus, we are unable to determine the safety and efficacy of gene therapy for haemophilia. Gene therapy for haemophilia is still in its nascent stages and there is a need for well-designed clinical trials to assess the long-term feasibility, success and risks of gene therapy for people with haemophilia.

Gene therapy for haemophilia.

BACKGROUND: Haemophilia is a genetic disorder which is characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft tissues. Current methods of treatment are expensive, challenging and involve regular administration of clotting factors. Gene therapy has recently been prompted as a curative treatment modality. OBJECTIVES: To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis & Genetic Disorders Group's Coagulopathies Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. We also searched the reference lists of relevant articles and reviews.Date of last search: 06 November 2014. SELECTION CRITERIA: Eligible trials included randomised or quasi-randomised clinical trials, including controlled clinical trials comparing gene therapy (with or without standard treatment) with standard treatment (factor replacement) or other 'curative' treatment such as stem cell transplantation individuals with haemophilia A or B of all ages who do not have inhibitors to factor VIII or IX. DATA COLLECTION AND ANALYSIS: No trials of gene therapy for haemophilia were found. MAIN RESULTS: No trials of gene therapy for haemophilia were identified. AUTHORS' CONCLUSIONS: No randomised or quasi-randomised clinical trials of gene therapy for haemophilia were identified. Thus, we are unable to determine the effects of gene therapy for haemophilia. Gene therapy for haemophilia is still in its nascent stages and there is a need for well-designed clinical trials to assess the long-term feasibility, success and risks of gene therapy for people with haemophilia.

Using PubMed in radiology: Ten useful tips for radiologists.

PubMed contains a bibliography of articles published in around 4800 journals. It combines MEDLINE and OLDMEDLINE (articles from 1960, going back till the 1940s). PubMed is updated on a daily basis; to include both published and ahead of print references. As a radiologist, one can use PubMed to track several journals, track topics, search for specific topics, verify incomplete or incorrect references, store one's own publications, and save selected references; one can also create filters depending on one's own search needs for some regular topics. This article provides some key background knowledge on searching PubMed and also describes some features that are often left unexplored. The PubMed site has undergone many changes in the last few years and this article will update users on the current features.

Information retrieval for dermatologists--an introduction.

The use of the internet in all fields of medicine is becoming more and more common in most areas of the world. The factors contributing to limited or no usage are difficulty of access due to poor IT infrastructure, paucity of time and lack of awareness and knowledge of the optimal use of the internet. For healthcare professionals, lack of time is one of the most common reasons for venturing into anything new. The only way to tackle this is to make a conscious commitment to learning how to use the Internet effectively. The least that one could strive to do is to learn enough to get internet usage handled by junior colleagues, secretaries and external resources, and expect the best of outputs from each. This article will focus on the internet for dermatologists. It will cover a range of resources and include search techniques and evaluation criteria.