Establishment of a web‐based registry for rare (orphan) pediatric lung diseases in the United Kingdom: The BPOLD registry

Laverty, Aidan; Jaffé, Adam; Cunningham, Steve

doi:10.1002/ppul.20783

Cited by 33 publications

(38 citation statements)

References 15 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…This gap is increasingly addressed by clinicians through the establishment of research networks and registries, including the British Paediatric Orphan Lung Disease registry,44 the UK Obstetric Surveillance System,45 and the InterRett database 46. The Rare Diseases Clinical Research Network35 and Orphanet34 facilitate the development of registries, national centres of reference and research networks.…”

Section: Resultsmentioning

confidence: 99%

Rare childhood diseases: how should we respond?

Zurynski

Frith

Leonard

et al. 2008

Archives of Disease in Childhood

120

119

View full text Add to dashboard Cite

Paradoxically, rare diseases are common, collectively affecting 6-10% of the population and have a huge impact on patients and families, health services, clinicians and the wider community. Accurate data are required to inform clinical practice, government policy and health service planning. We recommend a national approach, similar to that adopted in the USA and Europe, to support research and promote advocacy and equitable access to services for children with rare diseases.

show abstract

Section: Resultsmentioning

confidence: 99%

Rare childhood diseases: how should we respond?

Zurynski

Frith

Leonard

et al. 2008

Archives of Disease in Childhood

120

119

View full text Add to dashboard Cite

show abstract

“…In addition, patient registries, such as the British Pediatric Orphan Lung Disease Registry (www.bpold.-co.uk), afford an opportunity to increase our understanding of these rare diseases by accumulating a data set on children with cystic lung lesions. 38 …”

Section: Introductionmentioning

confidence: 99%

Pulmonary cysts in early childhood and the risk of malignancy

Priest

Williams

Hill

et al. 2008

Pediatric Pulmonology

264

215

View full text Add to dashboard Cite

Summary. Surgery for congenital and early childhood lung cysts is often dictated by symptoms such as respiratory distress, infection or pneumothorax. Asymptomatic cysts present a therapeutic dilemma: surgical intervention and ''conservative'' observation have advocates. The risk of malignancy in such cysts is considered by some an indication for surgical intervention and is reviewed in this paper. Pleuropulmonary blastoma (PPB) is the most frequent malignancy associated with childhood lung cysts. Although rare, PPB occurs predictably in certain clinical and familial situations. This unique biology of PPB can inform the cyst management decision. The earliest manifestation of PPB is a malignant lung cyst in young children, clinically and radiographically indistinguishable from benign congenital lung cysts. Histopathologic examination differentiates cystic PPB from the benign cystic variants. Surgical excision of cystic PPB (with or without chemotherapy) cures approximately 85-90% of children. If not excised, cystic PPB evolves to cystic/solid or solid high-grade sarcoma (cure rate 45-60%) by age 2-6 years. Numerous reports of ''malignancy in a congenital lung cyst'' are now understood as the characteristic progression of cystic PPB. PPB is genetically determined in many cases. Detailed family history may reveal the hallmarks of PPB in the patient or young relatives: a unique constellation of diseases including lung cysts, cystic nephroma, childhood cancers, stromal sex-chord ovarian tumors, seminomas or dysgerminomas, intestinal polyps, thyroid hyperplasias, and hamartomas. Pneumothorax and multifocal/bilateral lung cysts also characterize PPB. These diagnoses predict that a lung cyst is more likely PPB than a benign congenital cyst. Patients fitting this pattern deserve histologic diagnosis. The genetic basis for this heritable syndrome is unknown but is being actively investigated.

show abstract

“…Although several specialty epidemiological surveillance systems have adopted electronic card reporting,8 this is the first system we are aware of that has been used by a specific group of physicians to actively and regularly report ADRs 3. Clarkson et al have shown paediatricians are willing to report but this has never been activated on a national scale 4.…”

Section: Discussionmentioning

confidence: 99%

The use of electronic reporting to aid surveillance of adverse drug reactions (ADRs) in children: a proof of concept study

Lynn

Riding

McIntosh

2010

Archives of Disease in Childhood

View full text Add to dashboard Cite

Objective To investigate: (1) the feasibility of establishing active paediatric surveillance for adverse drug reactions (ADRs), (2) whether electronic reporting is effective and (3) whether such a system could complement the Medicines and Healthcare products Regulatory Agency (MHRA) yellow card system. Design Between January 2006 and February 2007 ADRs in children under 16 were reported each month by consultant paediatricians and paediatric pharmacists in Scotland. For 8 months respondents received a postal card, after which half were selected to report electronically via an email card for a further 6 months and half continued with the postal card. Reports of paediatric ADRs severe enough to warrant hospital admission or to delay discharge of hospitalised patients or resulting from an outpatient prescription were followed up. A postal questionnaire evaluated the system at the end of the study. Results Following a 2-month lead-in period, 83% of the cards were returned over the year, 84% by paediatricians and 82% by pharmacists. With electronic reporting the response rate was 80%. Eighty-seven confi rmed reports of a drug being associated with one or more adverse reactions were reported in 67 children. Only eight children were also identifi ed through the MHRA yellow card system. Respondents indicated continuing willingness to contribute to an active (preferably electronic) reporting system. Conclusions Active paediatric ADR surveillance is feasible; electronic reporting is effective and data collected are different to, but could complement, those collected by the MHRA yellow card.

show abstract

Establishment of a web‐based registry for rare (orphan) pediatric lung diseases in the United Kingdom: The BPOLD registry

Cited by 33 publications

References 15 publications

Rare childhood diseases: how should we respond?

Rare childhood diseases: how should we respond?

Pulmonary cysts in early childhood and the risk of malignancy

The use of electronic reporting to aid surveillance of adverse drug reactions (ADRs) in children: a proof of concept study

Contact Info

Product

Resources

About