Literature DB >> 22544437

Newborn screening programmes in Europe; arguments and efforts regarding harmonization. Part 2. From screening laboratory results to treatment, follow-up and quality assurance.

Peter Burgard1, Kathrin Rupp, Martin Lindner, Gisela Haege, Tessel Rigter, Stephanie S Weinreich, J Gerard Loeber, Domenica Taruscio, Luciano Vittozzi, Martina C Cornel, Georg F Hoffmann.   

Abstract

In a survey conducted in 2010/2011 data from the 28 EU member states, four EU candidate states (Croatia, FYROM, Iceland, Turkey), three potential EU candidate states (Bosnia Herzegovina, Montenegro, Serbia), and two EFTA states (Norway and Switzerland) were collected. The status and function of newborn screening (NBS) programmes were investigated from the information to prospective parents and the public via confirmation of a positive screening result up to decisions on treatment. This article summarises the results from screening laboratory findings to start of treatment. In addition we asked about the existence of feedback loops reporting the conclusions of confirmation of screening results to the screening laboratory and communication of long-term outcome to diagnostic units and possibly existing central registries. Parallel to the description of actual practices of where, how and by whom the different steps of the programmes are executed, we also asked for the existence of guidelines or directives regulating the screening programmes, material to support information of parents about diagnoses and treatment and training facilities for professionals involved in the programmes. This survey gives a first comprehensive overview of the steps following a positive screening result in European NBS programmes. The 37 data sets reveal substantial variation of national screening panels, but also a lot of similarities. Analysis across all countries revealed that actual practice is often organised but not regulated by guidelines. Material to inform patients is available more often for explaining treatment (69 %) than explaining the necessity of confirmatory diagnostics (41 %). Training of professionals is rarely regulated by a guideline (2 %), but is offered for paediatricians (40 %) and dieticians (29 %) and only rarely for other professions (e.g. geneticists, clinical nurse specialists, psychologists). Registry-based evaluation of long-term outcome is as yet almost nonexistent (3 %).

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Year:  2012        PMID: 22544437     DOI: 10.1007/s10545-012-9484-z

Source DB:  PubMed          Journal:  J Inherit Metab Dis        ISSN: 0141-8955            Impact factor:   4.982


  16 in total

1.  Use of guidelines improves the neurological outcome in glutaric aciduria type I.

Authors:  Jana Heringer; S P Nikolas Boy; Regina Ensenauer; Birgit Assmann; Johannes Zschocke; Inga Harting; Thomas Lücke; Esther M Maier; Chris Mühlhausen; Gisela Haege; Georg F Hoffmann; Peter Burgard; Stefan Kölker
Journal:  Ann Neurol       Date:  2010-11       Impact factor: 10.422

2.  Completeness and complexity of information available to parents from newborn-screening programs.

Authors:  Kathryn E Fant; Sarah J Clark; Alex R Kemper
Journal:  Pediatrics       Date:  2005-05       Impact factor: 7.124

3.  A look at newborn screening: today and tomorrow.

Authors:  Peter C van Dyck; E Stephen Edwards
Journal:  Pediatrics       Date:  2006-05       Impact factor: 7.124

4.  Introducing new screens: why are we all doing different things?

Authors:  R J Pollitt
Journal:  J Inherit Metab Dis       Date:  2007-07-06       Impact factor: 4.982

Review 5.  Newborn screening: how are we travelling, and where should we be going?

Authors:  Bridget Wilcken
Journal:  J Inherit Metab Dis       Date:  2011-04-16       Impact factor: 4.982

6.  Natural history, outcome, and treatment efficacy in children and adults with glutaryl-CoA dehydrogenase deficiency.

Authors:  Stefan Kölker; Sven F Garbade; Cheryl R Greenberg; James V Leonard; Jean-Marie Saudubray; Antonia Ribes; H Serap Kalkanoglu; Allan M Lund; Begoña Merinero; Moacir Wajner; Mónica Troncoso; Monique Williams; John H Walter; Jaume Campistol; Milagros Martí-Herrero; Melissa Caswill; Alberto B Burlina; Florian Lagler; Esther M Maier; Bernd Schwahn; Aysegul Tokatli; Ali Dursun; Turgay Coskun; Ronald A Chalmers; David M Koeller; Johannes Zschocke; Ernst Christensen; Peter Burgard; Georg F Hoffmann
Journal:  Pediatr Res       Date:  2006-04-26       Impact factor: 3.756

Review 7.  Number needed to screen: development of a statistic for disease screening.

Authors:  C M Rembold
Journal:  BMJ       Date:  1998-08-01

8.  Revisiting the Wilson-Jungner criteria: how can supplemental criteria guide public health in the era of genetic screening?

Authors:  Michael Petros
Journal:  Genet Med       Date:  2011-10-07       Impact factor: 8.822

9.  Guidelines for the Development of Comprehensive Care Centers for Congenital Adrenal Hyperplasia: Guidance from the CARES Foundation Initiative.

Authors:  Richard J Auchus; Selma Feldman Witchel; Kelly R Leight; Javier Aisenberg; Ricardo Azziz; Tânia A Bachega; Linda A Baker; Arlene B Baratz; Laurence S Baskin; Sheri A Berenbaum; David T Breault; Barbara I Cerame; Gerard S Conway; Erica A Eugster; Stephanie Fracassa; John P Gearhart; Mitchell E Geffner; Katharine B Harris; Richard S Hurwitz; Aviva L Katz; Brinda N Kalro; Peter A Lee; Gretchen Alger Lin; Karen J Loechner; Ian Marshall; Deborah P Merke; Claude J Migeon; Walter L Miller; Tamara L Nenadovich; Sharon E Oberfield; Kenneth A Pass; Dix P Poppas; Michele A Lloyd-Puryear; Charmian A Quigley; Felix G Riepe; Richard C Rink; Scott A Rivkees; David E Sandberg; Traci L Schaeffer; Richard N Schlussel; Francis X Schneck; Ellen W Seely; Diane Snyder; Phyllis W Speiser; Bradford L Therrell; Carol Vanryzin; Maria G Vogiatzi; Michael P Wajnrajch; Perrin C White; Alan E Zuckerman
Journal:  Int J Pediatr Endocrinol       Date:  2011-01-10

Review 10.  Diagnosis and management of glutaric aciduria type I--revised recommendations.

Authors:  Stefan Kölker; Ernst Christensen; James V Leonard; Cheryl R Greenberg; Avihu Boneh; Alberto B Burlina; Alessandro P Burlina; Marjorie Dixon; Marinus Duran; Angels García Cazorla; Stephen I Goodman; David M Koeller; Mårten Kyllerman; Chris Mühlhausen; Edith Müller; Jürgen G Okun; Bridget Wilcken; Georg F Hoffmann; Peter Burgard
Journal:  J Inherit Metab Dis       Date:  2011-03-23       Impact factor: 4.982

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  29 in total

Review 1.  Newborn bloodspot screening policy framework for Australia.

Authors:  Peter O'Leary; Susannah Maxwell
Journal:  Australas Med J       Date:  2015-09-30

2.  The phenotypic spectrum of organic acidurias and urea cycle disorders. Part 1: the initial presentation.

Authors:  Stefan Kölker; Angeles Garcia-Cazorla; Angeles Garcia Cazorla; Vassili Valayannopoulos; Allan M Lund; Alberto B Burlina; Jolanta Sykut-Cegielska; Frits A Wijburg; Elisa Leão Teles; Jiri Zeman; Carlo Dionisi-Vici; Ivo Barić; Daniela Karall; Persephone Augoustides-Savvopoulou; Lise Aksglaede; Jean-Baptiste Arnoux; Paula Avram; Matthias R Baumgartner; Javier Blasco-Alonso; Brigitte Chabrol; Anupam Chakrapani; Kimberly Chapman; Elisenda Cortès I Saladelafont; Maria L Couce; Linda de Meirleir; Dries Dobbelaere; Veronika Dvorakova; Francesca Furlan; Florian Gleich; Wanda Gradowska; Stephanie Grünewald; Anil Jalan; Johannes Häberle; Gisela Haege; Robin Lachmann; Alexander Laemmle; Eveline Langereis; Pascale de Lonlay; Diego Martinelli; Shirou Matsumoto; Chris Mühlhausen; Hélène Ogier de Baulny; Carlos Ortez; Luis Peña-Quintana; Danijela Petković Ramadža; Esmeralda Rodrigues; Sabine Scholl-Bürgi; Etienne Sokal; Christian Staufner; Marshall L Summar; Nicholas Thompson; Roshni Vara; Inmaculada Vives Pinera; John H Walter; Monique Williams; Peter Burgard
Journal:  J Inherit Metab Dis       Date:  2015-04-15       Impact factor: 4.982

3.  The development and organization of newborn screening programs in Turkey.

Authors:  Başak Tezel; Dilek Dilli; Hilal Bolat; Hatice Sahman; Sema Ozbaş; Deniz Acıcan; Mustafa Ertek; Mehmet Rıfat Köse; Uğur Dilmen
Journal:  J Clin Lab Anal       Date:  2013-12-27       Impact factor: 2.352

4.  Considering consent: a structural equation modelling analysis of factors influencing decisional quality when accepting newborn screening.

Authors:  Stuart G Nicholls; Kevin W Southern
Journal:  J Inherit Metab Dis       Date:  2013-09-17       Impact factor: 4.982

5.  Age at disease onset and peak ammonium level rather than interventional variables predict the neurological outcome in urea cycle disorders.

Authors:  Roland Posset; Angeles Garcia-Cazorla; Vassili Valayannopoulos; Elisa Leão Teles; Carlo Dionisi-Vici; Anaïs Brassier; Alberto B Burlina; Peter Burgard; Elisenda Cortès-Saladelafont; Dries Dobbelaere; Maria L Couce; Jolanta Sykut-Cegielska; Johannes Häberle; Allan M Lund; Anupam Chakrapani; Manuel Schiff; John H Walter; Jiri Zeman; Roshni Vara; Stefan Kölker
Journal:  J Inherit Metab Dis       Date:  2016-04-22       Impact factor: 4.982

6.  Clinical practice: experience with newborn screening for congenital hypothyroidism in the Republic of Macedonia - a multiethnic country.

Authors:  Mirjana Kocova; Violeta Anastasovska; Elena Sukarova-Angelovska; Milica Tanaskoska; Elizabeta Taseva
Journal:  Eur J Pediatr       Date:  2014-09-07       Impact factor: 3.183

Review 7.  The Italian National Centre for Rare Diseases: where research and public health translate into action.

Authors:  Domenica Taruscio; Linda Agresta; Annalisa Amato; Giuseppe Bernardo; Luana Bernardo; Francesca Braguti; Pietro Carbone; Claudio Carta; Marina Ceccarini; Federica Censi; Simona Coppola; Patrizia Crialese; Marta De Santis; Stefano Diemoz; Carlo Donati; Sabina Gainotti; Gianluca Ferrari; Giovanna Floridia; Claudio Frank; Rosa Giuseppa Frazzica; Amalia E Gentile; Orietta Granata; Yllka Kodra; Manuela Latrofa; Paola Laricchiuta; Armando Magrelli; Cristina Morciano; Agata Polizzi; Stefania Razeto; Marco Salvatore; Antonella Sanseverino; Daniele Savini; Paola Torreri; Fabrizio Tosto; Flavia Villani; Giorgio Vincenti; Luciano Vittozzi
Journal:  Blood Transfus       Date:  2014-04       Impact factor: 3.443

8.  Newborn Screening Programmes in Europe, Arguments and Efforts Regarding Harmonisation: Focus on Organic Acidurias.

Authors:  Friederike Hörster; Stefan Kölker; J Gerard Loeber; Martina C Cornel; Georg F Hoffmann; Peter Burgard
Journal:  JIMD Rep       Date:  2016-06-26

9.  Neonatal Screening for Congenital Metabolic and Endocrine Disorders–Results From Germany for the Years 2006–2018.

Authors:  Anja Lüders; Oliver Blankenstein; Inken Brockow; Regina Ensenauer; Martin Lindner; Andreas Schulze; Uta Nennstiel
Journal:  Dtsch Arztebl Int       Date:  2021-02-19       Impact factor: 5.594

10.  Newborn Screening: Still Room for Improvement.

Authors:  Klaus-Peter Zimmer
Journal:  Dtsch Arztebl Int       Date:  2021-02-19       Impact factor: 5.594

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