Sickle-cell Disease Registry of the GPOH

NCT03327428 | Recruiting

• 1 other identifier: Register Sichelzellkrankheit
• Study Type: observational
• Target: 1,000
• Locations: 1 country
• Sites: 1

Brief Summary

Sickle cell disease is one of the most common hereditary diseases. Most severe complications can be avoided if the disease is detected early and treated appropriately. The sickle cell disease registry of the Society for Paediatric Oncology/Haematology aims at describing the epidemiology of sickle cell disease in German-speaking central Europe. Patients with sickle cell disease will be characterized clinically and genetically and treatment will be documented with the aim to find predictors of the course of disease. In addition, the registry results should provide a solid evidence base to incorporate sickle cell disease into routine newborn screening and to update the national guidelines for the management of patients suffering from sickle cell disease in Germany. A consortium of five university hospitals (Berlin, Frankfurt, Hamburg, Heidelberg, Ulm) has been mandated by the Society for Paediatric Oncology/Haematology to implement this registry. The number of participating centers is constantly increasing and new centers that take care of either pediatric or adult patients with sickle cell disease are encouraged to support the registry. For further information please refer to: http://www.sichelzellkrankheit.info/

Conditions

Sickle Cell Disease

Keywords

Anemia, Sickle Cell; Sickle Cell disease

Outcome Measures

Primary Outcomes (1)

• Change in incidence of sickle-cell disease

  The incidence of sickle-cell disease will be reported every year in comparison to the preceding Report.

  Baseline and yearly, up to 10 years

Secondary Outcomes (2)

• Complications of sickle-cell disease

  Baseline and yearly, up to 10 years

• Treatment of sickle-cell disease

  Baseline and yearly, up to 10 years

Study Arms (1)

Patients with Sickle Cell Disease

Patients with any sickling condition, including among others Sickle Cell Anemia, HbSC Disease, HbS-betaThal, excluding Sickle Cell Trait.

Eligibility Criteria

• Age: 0 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All patients with sickle cell disease being treated at participating centers that signed the informed consent form.

You may qualify if:

• signed informed consent
• current residency in either Germany, Austria or Switzerland
• sickle cell disease confirmed by hemoglobin analysis or molecular genetic analysis
• Homozygous sickle cell disease (HbSS)
• HbSC disease
• Sickle cell disease HbS / bThal
• Other, rare sickle cell syndromes such as HbS/OArab, HbS/HPFH, HbS/E, HbS/D Punjab, HbS/C Harlem, HbC/S Antilles, HbS/Quebec-CHORI, HbA/S Oman, HbA/Jamaica Plain

You may not qualify if:

• \- isolated heterozygous trait for HbS

Sponsors & Collaborators

• University Hospital Heidelberg: lead
• GPOH Consortium Sickle Cell Disease: collaborator
• Johann Wolfgang Goethe University Hospital: collaborator
• Universitätsklinikum Hamburg-Eppendorf: collaborator
• University Hospital Ulm: collaborator
• Charite University, Berlin, Germany: collaborator
• German Cancer Research Center: collaborator
• Deutsche Kinderkrebsstiftung: collaborator
• Pfizer: collaborator

Study Sites (1)

Center for Child and Adolescent Medicine, University Medical Center Heidelberg

Heidelberg, Baden-Wurttemberg, 69124, Germany

RECRUITING

Related Publications (9)

• Lobitz S. Neugeborenenscreening auf Sichelzellkrankheiten in Deutschland. Kinder- und Jugendmedizin 2017;17(2):82-86 [German]

  BACKGROUND

• Kunz JB, Cario H, Grosse R, Jarisch A, Lobitz S, Kulozik AE. The epidemiology of sickle cell disease in Germany following recent large-scale immigration. Pediatr Blood Cancer. 2017 Jul;64(7). doi: 10.1002/pbc.26550. Epub 2017 Apr 6.

  PMID: 28383793 BACKGROUND

• Kunz JB, Awad S, Happich M, Muckenthaler L, Lindner M, Gramer G, Okun JG, Hoffmann GF, Bruckner T, Muckenthaler MU, Kulozik AE. Significant prevalence of sickle cell disease in Southwest Germany: results from a birth cohort study indicate the necessity for newborn screening. Ann Hematol. 2016 Feb;95(3):397-402. doi: 10.1007/s00277-015-2573-y. Epub 2015 Dec 12.

  PMID: 26658910 BACKGROUND

• Grosse R, Lukacs Z, Cobos PN, Oyen F, Ehmen C, Muntau B, Timmann C, Noack B. The Prevalence of Sickle Cell Disease and Its Implication for Newborn Screening in Germany (Hamburg Metropolitan Area). Pediatr Blood Cancer. 2016 Jan;63(1):168-70. doi: 10.1002/pbc.25706. Epub 2015 Aug 14.

  PMID: 26275168 BACKGROUND

• Frommel C, Brose A, Klein J, Blankenstein O, Lobitz S. Newborn screening for sickle cell disease: technical and legal aspects of a German pilot study with 38,220 participants. Biomed Res Int. 2014;2014:695828. doi: 10.1155/2014/695828. Epub 2014 Jul 23.

  PMID: 25147811 BACKGROUND

• Lobitz S, Frommel C, Brose A, Klein J, Blankenstein O. Incidence of sickle cell disease in an unselected cohort of neonates born in Berlin, Germany. Eur J Hum Genet. 2014 Aug;22(8):1051-3. doi: 10.1038/ejhg.2013.286. Epub 2014 Jan 8.

  PMID: 24398797 BACKGROUND

• Kunz JB, Lobitz S, Grosse R, Oevermann L, Hakimeh D, Jarisch A, Cario H, Beier R, Schenk D, Schneider D, Gross-Wieltsch U, Prokop A, Heine S, Khurana C, Erlacher M, Durken M, Linke C, Fruhwald M, Corbacioglu S, Claviez A, Metzler M, Ebinger M, Full H, Wiesel T, Eberl W, Reinhard H, Tagliaferri L, Allard P, Karapanagiotou-Schenkel I, Rother LM, Beck D, Le Cornet L, Kulozik AE; German Sickle Cell Disease Registry. Sickle cell disease in Germany: Results from a national registry. Pediatr Blood Cancer. 2020 Apr;67(4):e28130. doi: 10.1002/pbc.28130. Epub 2019 Dec 22.

  PMID: 31867835 RESULT

• Kunz JB, Schlotmann A, Daubenbuchel A, Lobitz S, Jarisch A, Grosse R, Cario H, Oevermann L, Hakimeh D, Tagliaferri L, Kulozik AE. Benefits of a Disease Management Program for Sickle Cell Disease in Germany 2011-2019: The Increased Use of Hydroxyurea Correlates with a Reduced Frequency of Acute Chest Syndrome. J Clin Med. 2021 Sep 30;10(19):4543. doi: 10.3390/jcm10194543.

  PMID: 34640578 RESULT

• Allard P, Alhaj N, Lobitz S, Cario H, Jarisch A, Grosse R, Oevermann L, Hakimeh D, Tagliaferri L, Kohne E, Kopp-Schneider A, Kulozik AE, Kunz JB. Genetic modifiers of fetal hemoglobin affect the course of sickle cell disease in patients treated with hydroxyurea. Haematologica. 2022 Jul 1;107(7):1577-1588. doi: 10.3324/haematol.2021.278952.

  PMID: 34706496 RESULT

Related Links

• Official website of the Registry \[German\]
• Sickle Cell Disease Guidelines, German language

MeSH Terms

Conditions

Anemia, Sickle Cell

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

Study Officials

• Joachim Kunz, Dr.

  Center for Child and Adolescent Medicine, University Medical Center Heidelberg

  PRINCIPAL INVESTIGATOR

• Holger Cario, Prof. Dr.

  University Hospital Ulm

• Regine Grosse, Dr.

• Andrea Jarisch, Dr.

  Johann Wolfgang Goethe University Hospital

• Andreas Kulozik, Prof. Dr.

  University Hospital Heidelberg

• Stephan Lobitz, Dr. MSc

  Gemeinschaftsklinikum Mittelrhein, Koblenz

• Lena Oevermann

  Charite University, Berlin, Germany

Central Study Contacts

Joachim Kunz, Dr.

CONTACT

06221 56 4555; Joachim.Kunz@med.uni-heidelberg.de

Laura Tagliaferri, Dr.

CONTACT

06221 56 4555; Laura.Tagliaferri@med.uni-heidelberg.de

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 3 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Senior physician

Study Record Dates

• First Submitted: August 16, 2017
• First Posted: October 31, 2017
• Study Start: December 15, 2016
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2040
• Last Updated: January 24, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)