Sickle-cell Disease Registry of the GPOH

NCT ID: NCT03327428

Last Updated: 2025-01-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-12-15

Study Completion Date

2040-12-31

Brief Summary

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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/

Detailed Description

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Conditions

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Sickle Cell Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Patients with Sickle Cell Disease

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

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

\- isolated heterozygous trait for HbS
Minimum Eligible Age

0 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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GPOH Consortium Sickle Cell Disease

OTHER

Sponsor Role collaborator

Johann Wolfgang Goethe University Hospital

OTHER

Sponsor Role collaborator

Universitätsklinikum Hamburg-Eppendorf

OTHER

Sponsor Role collaborator

University Hospital Ulm

OTHER

Sponsor Role collaborator

Charite University, Berlin, Germany

OTHER

Sponsor Role collaborator

German Cancer Research Center

OTHER

Sponsor Role collaborator

Deutsche Kinderkrebsstiftung

OTHER

Sponsor Role collaborator

Pfizer

INDUSTRY

Sponsor Role collaborator

University Hospital Heidelberg

OTHER

Sponsor Role lead

Responsible Party

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Dr. Joachim Kunz

Senior physician

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Joachim Kunz, Dr.

Role: PRINCIPAL_INVESTIGATOR

Center for Child and Adolescent Medicine, University Medical Center Heidelberg

Holger Cario, Prof. Dr.

Role:

University Hospital Ulm

Regine Grosse, Dr.

Role:

Andrea Jarisch, Dr.

Role:

Johann Wolfgang Goethe University Hospital

Andreas Kulozik, Prof. Dr.

Role:

University Hospital Heidelberg

Stephan Lobitz, Dr. MSc

Role:

Gemeinschaftsklinikum Mittelrhein, Koblenz

Lena Oevermann

Role:

Charite University, Berlin, Germany

Locations

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Center for Child and Adolescent Medicine, University Medical Center Heidelberg

Heidelberg, Baden-Wurttemberg, Germany

Site Status RECRUITING

Countries

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Germany

Central Contacts

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Joachim Kunz, Dr.

Role: CONTACT

06221 56 4555

Laura Tagliaferri, Dr.

Role: CONTACT

06221 56 4555

Facility Contacts

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Joachim Kunz, Dr.

Role: primary

06221 56 4555

Laura Tagliaferri, Dr.

Role: backup

06221 56 4555

References

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

Reference Type 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.

Reference Type BACKGROUND
PMID: 28383793 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 26658910 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 26275168 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 25147811 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 24398797 (View on PubMed)

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.

Reference Type RESULT
PMID: 31867835 (View on PubMed)

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.

Reference Type RESULT
PMID: 34640578 (View on PubMed)

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.

Reference Type RESULT
PMID: 34706496 (View on PubMed)

Related Links

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http://www.sichelzellkrankheit.info/

Official website of the Registry \[German\]

https://www.awmf.org/leitlinien/detail/ll/025-016.html

Sickle Cell Disease Guidelines, German language

Other Identifiers

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Register Sichelzellkrankheit

Identifier Type: -

Identifier Source: org_study_id

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