Natural History of Sickle Cell Disease

NCT00081523 | Recruiting

• 2 other identifiers: 04016104-H-0161
• Study Type: observational
• Target: 3,500
• Locations: 1 country
• Sites: 3

Brief Summary

This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated. Subjects will receive their general medical care outside the NIH and will be seen at our clinic or at CNHS with varying frequency. Subjects may be seen for multiple visits. Subjects may be asked to return for additional testing as needed. Clinical care for patients with sickle cell disease will be provided as appropriate through the Sickle Cell Clinic and the inpatient clinical center....

Conditions

Pain Crisis

Keywords

Hemoglobin; Acute Chest Syndrome; Treatment Options; Nitric Oxide; Pulmonary Hypertension; Natural History

Outcome Measures

Primary Outcomes (1)

• To gather, through clinical experience, information regarding the natural history, co-morbid conditions and outcomes, and complications relating to sickle cell disease and other hemolytic disorders in minority/ethnic patients

  Better characterization of the natural history of sickle cell disease

  ongoing

Study Arms (1)

Patients

Individuals with known or suspected sickle cell disease

Eligibility Criteria

• Age: 2 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study will be open to all eligible subjects based on inclusion and exclusion criteria and who provide informed consent. Patients may self-refer, be recruited through the NIH office of recruitment, and may include patients participating on other NIH IRB approved protocols. The study will also be open to all eligible pediatric patients receiving medical care for SCD at Children s National.

You may qualify if:

• Individuals with known or suspected sickle cell disease
• years of age and older
• Willing to provide informed consent or appropriate informed consent from parent or legal guardian
• Patients seen at sickle outpatient clinics at any one of the participating centers (CNHS or NIH).

You may not qualify if:

• Patient and/or guardian unable and unwilling to give informed consent or assent.
• Patients less than 2 years of age.

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead

Study Sites (3)

Childrens National Health Center

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Suburban Hospital

Bethesda, Maryland, 20814, United States

COMPLETED

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Publications (8)

• Parrow NL, Doherty JM, Conrey A, Thein SL, Fleming RE. Relationships Between Markers of Iron Status and Hematological Parameters in Patients With Sickle Cell Disease. Adv Hematol. 2024 Dec 3;2024:9872440. doi: 10.1155/ah/9872440. eCollection 2024.

  PMID: 39659429 DERIVED

• Tumburu L, Ghosh-Choudhary S, Seifuddin FT, Barbu EA, Yang S, Ahmad MM, Wilkins LHW, Tunc I, Sivakumar I, Nichols JS, Dagur PK, Yang S, Almeida LEF, Quezado ZMN, Combs CA, Lindberg E, Bleck CKE, Zhu J, Shet AS, Chung JH, Pirooznia M, Thein SL. Circulating mitochondrial DNA is a proinflammatory DAMP in sickle cell disease. Blood. 2021 Jun 3;137(22):3116-3126. doi: 10.1182/blood.2020009063.

  PMID: 33661274 DERIVED

• Giudice V, Feng X, Lin Z, Hu W, Zhang F, Qiao W, Ibanez MDPF, Rios O, Young NS. Deep sequencing and flow cytometric characterization of expanded effector memory CD8+CD57+ T cells frequently reveals T-cell receptor Vbeta oligoclonality and CDR3 homology in acquired aplastic anemia. Haematologica. 2018 May;103(5):759-769. doi: 10.3324/haematol.2017.176701. Epub 2018 Feb 1.

  PMID: 29419434 DERIVED

• Nguyen KL, Tian X, Alam S, Mehari A, Leung SW, Seamon C, Allen D, Minniti CP, Sachdev V, Arai AE, Kato GJ. Elevated transpulmonary gradient and cardiac magnetic resonance-derived right ventricular remodeling predict poor outcomes in sickle cell disease. Haematologica. 2016 Feb;101(2):e40-3. doi: 10.3324/haematol.2015.125229. Epub 2015 Nov 20. No abstract available.

  PMID: 26589907 DERIVED

• Wang X, Mendelsohn L, Rogers H, Leitman S, Raghavachari N, Yang Y, Yau YY, Tallack M, Perkins A, Taylor JG 6th, Noguchi CT, Kato GJ. Heme-bound iron activates placenta growth factor in erythroid cells via erythroid Kruppel-like factor. Blood. 2014 Aug 7;124(6):946-54. doi: 10.1182/blood-2013-11-539718. Epub 2014 Jun 10.

  PMID: 24916507 DERIVED

• Koh C, Turner T, Zhao X, Minniti CP, Feld JJ, Simpson J, Demino M, Conrey AK, Jackson MJ, Seamon C, Kleiner DE, Kato GJ, Heller T. Liver stiffness increases acutely during sickle cell vaso-occlusive crisis. Am J Hematol. 2013 Nov;88(11):E250-4. doi: 10.1002/ajh.23532. Epub 2013 Aug 1.

  PMID: 23828202 DERIVED

• Tumblin A, Tailor A, Hoehn GT, Mack AK, Mendelsohn L, Freeman L, Xu X, Remaley AT, Munson PJ, Suffredini AF, Kato GJ. Apolipoprotein A-I and serum amyloid A plasma levels are biomarkers of acute painful episodes in patients with sickle cell disease. Haematologica. 2010 Sep;95(9):1467-72. doi: 10.3324/haematol.2009.018044. Epub 2010 Apr 7.

  PMID: 20378559 DERIVED

• Olnes M, Chi A, Haney C, May R, Minniti C, Taylor J 6th, Kato GJ. Improvement in hemolysis and pulmonary arterial systolic pressure in adult patients with sickle cell disease during treatment with hydroxyurea. Am J Hematol. 2009 Aug;84(8):530-32. doi: 10.1002/ajh.21446.

  PMID: 19536844 DERIVED

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Acute Chest Syndrome; Hypertension, Pulmonary

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Respiration Disorders; Anemia, Sickle Cell; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hypertension; Vascular Diseases; Cardiovascular Diseases

Study Officials

• Swee Lay Thein, M.D.

  National Heart, Lung, and Blood Institute (NHLBI)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Nancy A Asomaning

CONTACT

(301) 605-0398; nancy.asomaning@nih.gov

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 15, 2004
• First Posted: April 15, 2004
• Study Start: April 29, 2004
• Last Updated: May 7, 2026

Record last verified: 2026-02-17

Locations

US (3)