ArticleOrphanet journal of rare diseases2013
Organizing national responses for rare blood disorders: the Italian experience with sickle cell disease in childhood.
Article in Orphanet journal of rare diseases, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
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Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 41 citations in OpenAlex.
- Second-line therapy in paediatric warm autoimmune haemolytic anaemia. Guidelines from the Associazione Italiana Onco-Ematologia Pediatrica (AIEOP).Blood transfusion = Trasfusione del sangue · 2018Guideline
- Chronic Hydroxyurea Therapy in Children with Sickle Cell Anemia: Mechanisms of Action, Systemic Effects, and Long-Term Safety.Journal of clinical medicine · 2025Review
- Oxford Hip and Shoulder Scores as Potential Tools for the Early Detection of Avascular Necrosis in Apparently Unaffected Sites in Sickle Cell Disease: Results from a Prospective Cohort Study.Journal of clinical medicine · 2025Article
- The phenotypes of sickle cell disease: strategies to aid the identification of undiagnosed patients in the Italian landscape.Italian journal of pediatrics · 2025Review
- Screening for sickle cell disease by point-of-care tests in Italy: pilot study on 1000 at risk children.European journal of pediatrics · 2025Article
- Sickle cell disease: suspect, check, diagnose-practical tips for non-SCD experts to suspect and diagnose SCD in low-prevalence European settings.Frontiers in medicine · 2025Review
- Long-term outcomes of avascular necrosis in sickle cell disease using joint-specific patient-reported outcome measures: Results from a multicentre study.British journal of haematology · 2025Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Pediatric Moyamoya Disease and Syndrome in Italy: A Multicenter Cohort.Frontiers in pediatrics · 2022Article
- Global geographic differences in healthcare utilization for sickle cell disease pain crises in the CASiRe cohort.Blood cells, molecules & diseases · 2021Article
- Acute events in children with sickle cell disease in Italy during the COVID-19 pandemic: useful lessons learned.British journal of haematology · 2021Article
- Disease burden and quality of life of in children with sickle cell disease in Italy: time to be considered a priority.Italian journal of pediatrics · 2021Review
- An Educational Study Promoting the Delivery of Transcranial Doppler Ultrasound Screening in Paediatric Sickle Cell Disease: A European Multi-Centre Perspective.Journal of clinical medicine · 2019Article
- Current challenges in the management of patients with sickle cell disease - A report of the Italian experience.Orphanet journal of rare diseases · 2019Review
- Newborn Screening for Sickle Cell Disease in Europe.International journal of neonatal screening · 2019Review
- Evaluation of Technical Issues in a Pilot Multicenter Newborn Screening Program for Sickle Cell Disease.International journal of neonatal screening · 2019Article
- Haploidentical stem cell transplantation cures autoimmune hepatitis and cerebrovascular disease in a patient with sickle cell disease.Bone marrow transplantation · 2018Article
- An Expert Review of Pharmacogenomics of Sickle Cell Disease Therapeutics: Not Yet Ready for Global Precision Medicine.Omics : a journal of integrative biology · 2016Review
Corrections and comments
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Authors and funding
9 authors at 6 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundSickle cell disease (SCD) is the most frequent hemoglobinopathy worldwide but remains a rare blood disorder in most western countries. Recommendations for standard of care have been produced in the United States, the United Kingdom and France, where this disease is relatively frequent because of earlier immigration from Africa. These recommendations have changed the clinical course of SCD but can be difficult to apply in other contexts. The Italian Association of Pediatric Hematology Oncology (AIEOP) decided to develop a common national response to the rising number of SCD patients in Italy with the following objectives: 1) to create a national working group focused on pediatric SCD, and 2) to develop tailored guidelines for the management of SCD that could be accessed and practiced by those involved in the care of children with SCD in Italy.
methodsGuidelines, adapted to the Italian social context and health system, were developed by 22 pediatric hematologists representing 54 AIEOP centers across Italy. The group met five times for a total of 128 hours in 22 months; documents and opinions were circulated via web.
resultsRecommendations regarding the prevention and treatment of the most relevant complications of SCD in childhood adapted to the Italian context and health system were produced.
conclusionsCreating a network of physicians involved in the day-to-day care of children with SCD is feasible in a country where it remains rare. Providing hematologists, primary and secondary care physicians, and caregivers across the country with web-based guidelines for the management of SCD tailored to the Italian context is the first step in building a sustainable response to a rare but emerging childhood blood disorder and in implementing the World Health Organization's suggestion "to design (and) implement … comprehensive national integrated programs for the prevention and management of SCD".
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