Transfusion Treatment in Patients With SCD

NCT03397017 | Completed

• 1 other identifier: SITE
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

Brief Summary

The "National Transfusion Treatment Survey in patients with sickle cell disease (SCD)" is a prospective longitudinal systemic study that was created in order to evaluate the therapeutic approach, mainly transfusional, in patients affected by SCD throughout Italy and to improve the quality of care and implement research. The survey will evaluate all patients affected by different forms of sickle cell disease (HbS homozygosis, Thalassoso-drepanocytosis, HbS / HbC compound heterozygosis, other possible genetic compounds). Patients will be selected according with a SCD diagnosis confirmed by standardized biochemical criteria or by DNA analysis. Patients will be excluded from the study who do not meet the these requirements, who are unable to understand the protocol or able to give informed consent in the absence of any legal representative. All data will be collected through a standard web-based application, which will be completed by the responsable investigator or by sub-investigators selected by each center, after registration on the site providing personal data and indicating the affiliation structure. All data will be subsequently encrypted by the Central Server. The operator will subsequently be able to access the patient's clinical data to perform the updates, in order to follow the patient's clinical evolution over time. The study will not involve any additional tests compared to the routine of patient control.

Conditions

Anemia, Sickle Cell

Outcome Measures

Primary Outcomes (1)

• Web-based recording of personal, therapy and complications data of patients with sickle cell anemia included in the National Transfusion Treatment Survey

  The Survey Data Sheet is divided into three parts: 1. Personal data 2. Therapy: * Chronic transfusion regimen * Acute transfusion regimen * Hydroxyurea 3. Complications: - * Alloimmunization * Transfusion reactions * Martial structure and iron-chelating therapy

  Data will be recorded up to six months

Interventions

Transfusion OTHER

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

All Survey participants will be recruited from the Centers distributed in Italy; inclusion in the study will be in compliance with the inclusion and exclusion criteria. The information and the manifestation of the Consent will be illustrated and, where necessary, discussed in the presence of a Cultural Linguistic Mediator.

You may qualify if:

• patients suffering of sickle cell disease, with diagnosis confirmed by standardized biochemical criteria or by matching mutations on globin genes by DNA analysis;
• all patients who consent to the study by signing the informed consent given by the U.O.C.

You may not qualify if:

• patients without a diagnosis confirmed according to standardized biochemical criteria or via mutation on globin genes by DNA analysis.
• all patients unable to understand the study protocol and to give informed consent and who have no legal representative.

Sponsors & Collaborators

• Società Italiana Talassemie ed Emoglobinopatie: lead
• Associazione Italiana de Ematologia e Oncologia Pediatrica: collaborator
• Società Italiana di Medicina Trasfusionale e Immunoematologia: collaborator

Study Sites (1)

Gian Luca Forni

Genova, Ge, 16121, Italy

Location

Related Publications (5)

• Ohene-Frempong K, Weiner SJ, Sleeper LA, Miller ST, Embury S, Moohr JW, Wethers DL, Pegelow CH, Gill FM. Cerebrovascular accidents in sickle cell disease: rates and risk factors. Blood. 1998 Jan 1;91(1):288-94.

  PMID: 9414296 BACKGROUND

• Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010 Dec 11;376(9757):2018-31. doi: 10.1016/S0140-6736(10)61029-X. Epub 2010 Dec 3.

  PMID: 21131035 BACKGROUND

• Piel FB, Tatem AJ, Huang Z, Gupta S, Williams TN, Weatherall DJ. Global migration and the changing distribution of sickle haemoglobin: a quantitative study of temporal trends between 1960 and 2000. Lancet Glob Health. 2014 Feb;2(2):e80-9. doi: 10.1016/S2214-109X(13)70150-5.

  PMID: 24748392 BACKGROUND

• Piel FB, Hay SI, Gupta S, Weatherall DJ, Williams TN. Global burden of sickle cell anaemia in children under five, 2010-2050: modelling based on demographics, excess mortality, and interventions. PLoS Med. 2013;10(7):e1001484. doi: 10.1371/journal.pmed.1001484. Epub 2013 Jul 16.

  PMID: 23874164 BACKGROUND

• Graziadei G, De Franceschi L, Sainati L, Venturelli D, Masera N, Bonomo P, Vassanelli A, Casale M, Lodi G, Voi V, Rigano P, Pinto VM, Quota A, Notarangelo LD, Russo G, Allo M, Rosso R, D'Ascola D, Facchini E, Macchi S, Arcioni F, Bonetti F, Rossi E, Sau A, Campisi S, Colarusso G, Giona F, Lisi R, Giordano P, Boscarol G, Filosa A, Marktel S, Maroni P, Murgia M, Origa R, Longo F, Bortolotti M, Colombatti R, Di Maggio R, Mariani R, Piperno A, Corti P, Fidone C, Palazzi G, Badalamenti L, Gianesin B, Piel FB, Forni GL. Transfusional Approach in Multi-Ethnic Sickle Cell Patients: Real-World Practice Data From a Multicenter Survey in Italy. Front Med (Lausanne). 2022 Mar 16;9:832154. doi: 10.3389/fmed.2022.832154. eCollection 2022.

  PMID: 35372393 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Blood Transfusion

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• Giovanna Graziadei, MD

  Fondazione IRCCS Policlinico di Milano con sede in Via Francesco Sforza 35, 20122 Milano

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2017
• First Posted: January 11, 2018
• Study Start: July 1, 2016
• Primary Completion: December 31, 2016
• Study Completion: June 30, 2022
• Last Updated: April 19, 2023

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)