Benefits and Harms of Treatment and Preventive Interventions for Hereditary Angioedema: Protocol for a Systematic Review and Network Meta-Analysis of Randomized Controlled Trials

Background: Hereditary angioedema (HAE) is a rare genetic disease that can lead to potentially life-threatening airway attacks. Although novel therapies for HAE treatment have become available over the past decades, a comparison of all available treatments has not yet been conducted. As such, we wil...

Full description

Bibliographic Details
Main Authors: Mati Chuamanochan, Sutthinee Phuprasertsak, Puncharas Weesasubpong, Chidchanok Ruengorn, Chabaphai Phosuya, Ratanaporn Awiphan, Brian Hutton, Kednapa Thavorn, Jonathan A. Bernstein, Surapon Nochaiwong
Format: Article
Language:English
Published: MDPI AG 2022-05-01
Series:Genes
Subjects:
Online Access:https://www.mdpi.com/2073-4425/13/5/924
_version_ 1797499591322501120
author Mati Chuamanochan
Sutthinee Phuprasertsak
Puncharas Weesasubpong
Chidchanok Ruengorn
Chabaphai Phosuya
Ratanaporn Awiphan
Brian Hutton
Kednapa Thavorn
Jonathan A. Bernstein
Surapon Nochaiwong
author_facet Mati Chuamanochan
Sutthinee Phuprasertsak
Puncharas Weesasubpong
Chidchanok Ruengorn
Chabaphai Phosuya
Ratanaporn Awiphan
Brian Hutton
Kednapa Thavorn
Jonathan A. Bernstein
Surapon Nochaiwong
author_sort Mati Chuamanochan
collection DOAJ
description Background: Hereditary angioedema (HAE) is a rare genetic disease that can lead to potentially life-threatening airway attacks. Although novel therapies for HAE treatment have become available over the past decades, a comparison of all available treatments has not yet been conducted. As such, we will perform a systematic review and network meta-analysis to identify the best evidence-based treatments for the management of acute attacks and prophylaxis of HAE. Methods: This study will include both parallel and crossover randomized controlled trials that have investigated prevention or treatment strategies for HAE attacks. We will search electronic databases, including Medline, Embase, PubMed, Cochrane Library, Scopus, and CINAHL, from inception with no language restrictions. Potential trials will be supplemented through a gray literature search. The process of study screening, selection, data extraction, risk-of-bias assessment, certainty assessment and classification of treatments will be performed independently by a pair of reviewers. Any discrepancy will be addressed through team discussion. A two-step approach of pairwise and network meta-analysis will be performed. The summarized effect estimates of direct and indirect treatment comparisons will be pooled using DerSimonion–Laird random-effects models. The incoherence assumption, in terms of the consistency of direct and indirect effects, will be assessed. An evidence-based synthesis will be performed, based on the magnitudes of effect size, evidence certainty, and ranking of treatment effects, with respect to treatment benefits and harms. Discussion: This systematic review and network meta-analysis will summarize evidence-based conclusions with respect to the ratio of benefits and harms arising from interventions for the treatment of acute attacks and prophylaxis of HAE. Evidence from this network estimate could promote the rational use of interventions among people living with HAE in clinical practice settings. PROSPERO registration number: CRD42021251367.
first_indexed 2024-03-10T03:49:36Z
format Article
id doaj.art-ba18007fb5984eb4bcbcb1d2f4be7ac2
institution Directory Open Access Journal
issn 2073-4425
language English
last_indexed 2024-03-10T03:49:36Z
publishDate 2022-05-01
publisher MDPI AG
record_format Article
series Genes
spelling doaj.art-ba18007fb5984eb4bcbcb1d2f4be7ac22023-11-23T11:11:40ZengMDPI AGGenes2073-44252022-05-0113592410.3390/genes13050924Benefits and Harms of Treatment and Preventive Interventions for Hereditary Angioedema: Protocol for a Systematic Review and Network Meta-Analysis of Randomized Controlled TrialsMati Chuamanochan0Sutthinee Phuprasertsak1Puncharas Weesasubpong2Chidchanok Ruengorn3Chabaphai Phosuya4Ratanaporn Awiphan5Brian Hutton6Kednapa Thavorn7Jonathan A. Bernstein8Surapon Nochaiwong9Division of Dermatology, Department of Internal Medicine, Faculty of Medicine, Chiang Mai University, Chiang Mai 50200, ThailandMaharaj Nakorn Chiang Mai Hospital, Chiang Mai 50200, ThailandPharmacoepidemiology and Statistics Research Center (PESRC), Chiang Mai University, Chiang Mai 50200, ThailandPharmacoepidemiology and Statistics Research Center (PESRC), Chiang Mai University, Chiang Mai 50200, ThailandPharmacoepidemiology and Statistics Research Center (PESRC), Chiang Mai University, Chiang Mai 50200, ThailandPharmacoepidemiology and Statistics Research Center (PESRC), Chiang Mai University, Chiang Mai 50200, ThailandOttawa Hospital Research Institute, Ottawa Hospital, Ottawa, ON K1H 8L6, CanadaPharmacoepidemiology and Statistics Research Center (PESRC), Chiang Mai University, Chiang Mai 50200, ThailandAllergy Section, Division of Immunology, Department of Internal Medicine, College of Medicine, University of Cincinnati, Cincinnati, OH 45267, USAPharmacoepidemiology and Statistics Research Center (PESRC), Chiang Mai University, Chiang Mai 50200, ThailandBackground: Hereditary angioedema (HAE) is a rare genetic disease that can lead to potentially life-threatening airway attacks. Although novel therapies for HAE treatment have become available over the past decades, a comparison of all available treatments has not yet been conducted. As such, we will perform a systematic review and network meta-analysis to identify the best evidence-based treatments for the management of acute attacks and prophylaxis of HAE. Methods: This study will include both parallel and crossover randomized controlled trials that have investigated prevention or treatment strategies for HAE attacks. We will search electronic databases, including Medline, Embase, PubMed, Cochrane Library, Scopus, and CINAHL, from inception with no language restrictions. Potential trials will be supplemented through a gray literature search. The process of study screening, selection, data extraction, risk-of-bias assessment, certainty assessment and classification of treatments will be performed independently by a pair of reviewers. Any discrepancy will be addressed through team discussion. A two-step approach of pairwise and network meta-analysis will be performed. The summarized effect estimates of direct and indirect treatment comparisons will be pooled using DerSimonion–Laird random-effects models. The incoherence assumption, in terms of the consistency of direct and indirect effects, will be assessed. An evidence-based synthesis will be performed, based on the magnitudes of effect size, evidence certainty, and ranking of treatment effects, with respect to treatment benefits and harms. Discussion: This systematic review and network meta-analysis will summarize evidence-based conclusions with respect to the ratio of benefits and harms arising from interventions for the treatment of acute attacks and prophylaxis of HAE. Evidence from this network estimate could promote the rational use of interventions among people living with HAE in clinical practice settings. PROSPERO registration number: CRD42021251367.https://www.mdpi.com/2073-4425/13/5/924complement 1 inhibitor deficiencyeffectivenessgenetic diseaseharmshereditary angioedemasystematic review
spellingShingle Mati Chuamanochan
Sutthinee Phuprasertsak
Puncharas Weesasubpong
Chidchanok Ruengorn
Chabaphai Phosuya
Ratanaporn Awiphan
Brian Hutton
Kednapa Thavorn
Jonathan A. Bernstein
Surapon Nochaiwong
Benefits and Harms of Treatment and Preventive Interventions for Hereditary Angioedema: Protocol for a Systematic Review and Network Meta-Analysis of Randomized Controlled Trials
Genes
complement 1 inhibitor deficiency
effectiveness
genetic disease
harms
hereditary angioedema
systematic review
title Benefits and Harms of Treatment and Preventive Interventions for Hereditary Angioedema: Protocol for a Systematic Review and Network Meta-Analysis of Randomized Controlled Trials
title_full Benefits and Harms of Treatment and Preventive Interventions for Hereditary Angioedema: Protocol for a Systematic Review and Network Meta-Analysis of Randomized Controlled Trials
title_fullStr Benefits and Harms of Treatment and Preventive Interventions for Hereditary Angioedema: Protocol for a Systematic Review and Network Meta-Analysis of Randomized Controlled Trials
title_full_unstemmed Benefits and Harms of Treatment and Preventive Interventions for Hereditary Angioedema: Protocol for a Systematic Review and Network Meta-Analysis of Randomized Controlled Trials
title_short Benefits and Harms of Treatment and Preventive Interventions for Hereditary Angioedema: Protocol for a Systematic Review and Network Meta-Analysis of Randomized Controlled Trials
title_sort benefits and harms of treatment and preventive interventions for hereditary angioedema protocol for a systematic review and network meta analysis of randomized controlled trials
topic complement 1 inhibitor deficiency
effectiveness
genetic disease
harms
hereditary angioedema
systematic review
url https://www.mdpi.com/2073-4425/13/5/924
work_keys_str_mv AT matichuamanochan benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials
AT sutthineephuprasertsak benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials
AT puncharasweesasubpong benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials
AT chidchanokruengorn benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials
AT chabaphaiphosuya benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials
AT ratanapornawiphan benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials
AT brianhutton benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials
AT kednapathavorn benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials
AT jonathanabernstein benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials
AT suraponnochaiwong benefitsandharmsoftreatmentandpreventiveinterventionsforhereditaryangioedemaprotocolforasystematicreviewandnetworkmetaanalysisofrandomizedcontrolledtrials