Sickle-cell Disease Registry of the GPOH (SichReg)

• ClinicalTrials.gov Identifier: NCT03327428
• Recruitment Status: Recruiting
• First Posted: October 31, 2017
• Last Update Posted: November 9, 2022
• Sponsor: University Hospital Heidelberg
• Collaborators: GPOH Consortium Sickle Cell Disease; Johann Wolfgang Goethe University Hospital; Universitätsklinikum Hamburg-Eppendorf; University Hospital Ulm; Charite University, Berlin, Germany; Deutsche Kinderkrebsstiftung
• Information provided by (Responsible Party): Dr. Joachim Kunz, University Hospital Heidelberg

Tracking Information

• First Submitted Date: August 16, 2017
• First Posted Date: October 31, 2017
• Last Update Posted Date: November 9, 2022
• Actual Study Start Date: December 15, 2016
• Estimated Primary Completion Date: December 31, 2026 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 26, 2017)

Change in incidence of sickle-cell disease [ Time Frame: Baseline and yearly, up to 10 years ]

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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: October 26, 2017)

• Complications of sickle-cell disease [ Time Frame: Baseline and yearly, up to 10 years ]
  In addition to the incidence of the disease itself also possible complications will be reported in comparison to the preceding report (in case of the first report, only the prevalence will be reported as baseline).
• Treatment of sickle-cell disease [ Time Frame: Baseline and yearly, up to 10 years ]
  In addition to the incidence of the disease itself also the treatment received will be reported in comparison to the preceding report (in case of the first report, only the prevalence will be reported as baseline).

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Sickle-cell Disease Registry of the GPOH
• Official Title: Register Sichelzellkrankheit Der GPOH

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/

• Detailed Description: Not Provided
• Study Type: Observational [Patient Registry]
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: 2 Years
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: All patients with sickle cell disease being treated at participating centers that signed the informed consent form.
• Condition: Sickle Cell Disease
• Intervention: Not Provided

Study Groups/Cohorts

Patients with Sickle Cell Disease

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

Publications *

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

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: October 26, 2017): 500
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 31, 2040
• Estimated Primary Completion Date: December 31, 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 0 Years to 100 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Joachim Kunz, Dr.; 06221 56 4555; Joachim.Kunz@med.uni-heidelberg.de

Contact: Laura Tagliaferri, Dr.; 06221 56 4555; Laura.Tagliaferri@med.uni-heidelberg.de

• Listed Location Countries: Germany

Administrative Information

• NCT Number: NCT03327428
• Other Study ID Numbers: Register Sichelzellkrankheit
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Dr. Joachim Kunz, University Hospital Heidelberg
• Original Responsible Party: Same as current
• Current Study Sponsor: University Hospital Heidelberg
• Original Study Sponsor: Same as current

Collaborators

• GPOH Consortium Sickle Cell Disease
• Johann Wolfgang Goethe University Hospital
• Universitätsklinikum Hamburg-Eppendorf
• University Hospital Ulm
• Charite University, Berlin, Germany
• Deutsche Kinderkrebsstiftung

Investigators

• Principal Investigator: Joachim Kunz, Dr.; Center for Child and Adolescent Medicine, University Medical Center Heidelberg
• Investigator: Holger Cario, Prof. Dr.; University Hospital Ulm
• Investigator: Regine Grosse, Dr.; Universitätsklinikum Hamburg-Eppendorf
• Investigator: Andrea Jarisch, Dr.; Johann Wolfgang Goethe University Hospital
• Investigator: Andreas Kulozik, Prof. Dr.; University Hospital Heidelberg
• Investigator: Stephan Lobitz, Dr. MSc; Gemeinschaftsklinikum Mittelrhein, Koblenz
• Investigator: Lena Oevermann; Charite University, Berlin, Germany

• PRS Account: University Hospital Heidelberg
• Verification Date: November 2022