This study is currently recruiting participants.
Number
000697-H
Sponsoring Institute
National Heart, Lung and Blood Institute (NHLBI)
Recruitment Detail
Type: Participants currently recruited/enrolled Gender: Male & Female Min Age: 18 Years Max Age: 65 Years
Referral Letter Required
Yes
Population Exclusion(s)
Children
Keywords
Chip; Immunosuppression; Malignant Neoplasms; Posttransplant Cyclophosphamide; Tp53 Mutations; Hydroxyurea-Treated Sickle Cell Disease; Natural History
Recruitment Keyword(s)
None
Condition(s)
Sickle Cell Disease; Pulmonary Disease; Renal Disease; Heart Disease
Investigational Drug(s)
Investigational Device(s)
Intervention(s)
Supporting Site
National Heart, Lung, and Blood Institute
Sickle cell disease (SCD) is an inherited blood disorder. It can damage the heart, lungs, and kidneys and can cause serious illness and death. Treatments such as bone marrow transplants and gene therapies can cure SCD; but, over time, these cures may increase the risk of organ damage and cancers. More research is needed to understand the long-term effects of treatments that cure SCD.
Objective:
To observe how different treatments for SCD affect the health of the heart, lungs, and kidneys over the long term.
Eligibility:
People aged 4 to 65 years with SCD who have been treated with either blood stem cell transplant or with standard disease-modifying therapy.
Design:
Participants will be followed in this natural history study for 5 years. Their annual visits to their regular doctors will serve as research study visits.
Participant s own doctors will conduct tests as part of their standard care. Researchers will collect data from these tests. Researchers may also review participants prior medical records.
Data to be used in research may come from these tests:
Physical exam. This may include heart rate, breathing rate, height, and weight.
Breathing tests (spirometry). These are given before and after a participant takes a rescue medication to see how well the lungs are functioning.
Heart tests (echocardiogram). These are images of the heart captured through sound waves.
Pulse oximetry. A sensor is placed on the finger to measure the amount of oxygen in the blood.
Participants will complete questionnaires. Topics will include their personal and family history, social environment, employment history, and education. At screening, they will be asked about their willingness to participate in research.
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INCLUSION CRITERIA: All Aims: Confirmed laboratory diagnosis of SCD; Ability to give informed consent (except for those who meet the criteria for a waiver of consent for the retrospective data cohort); Ability to provide pre- and post-curative therapy data to meet study aims Treated with either one HSCT (data collected after second curative therapy will be excluded),or with standard disease-modifying therapy. Aim 1a: Ages 4 to 17 years Aim 1b, 2, & 3: Ages 18 to 65 years Aim 3: Ages 4 to 65 years Study Population: Curative therapy cohort: Ages 4 - 65 years; receipt of HSCT or gene therapy/editing 2003 - 2026 at one of the 5 study sites. Retrospective: 2003 - 2021 Prospective: 2021 - 2026 Standard therapy control cohort: Ages 4 - 65 years; receipt of disease-modifying therapy only between 2003 - 2026; followed as part of the VUMC-based Tennessee Never Lost Cohort. EXCLUSION CRITERIA: Participants judged to be non-compliant based on previous experience in terms of clinic appointments and following advice.
All Aims:
Confirmed laboratory diagnosis of SCD;
Ability to give informed consent (except for those who meet the criteria for a waiver of consent for the retrospective data cohort);
Ability to provide pre- and post-curative therapy data to meet study aims
Treated with either one HSCT (data collected after second curative therapy will be excluded),or with standard disease-modifying therapy.
Aim 1a: Ages 4 to 17 years
Aim 1b, 2, & 3: Ages 18 to 65 years
Aim 3: Ages 4 to 65 years
Study Population:
Curative therapy cohort: Ages 4 - 65 years; receipt of HSCT or gene therapy/editing 2003 - 2026 at one of the 5 study sites.
Retrospective: 2003 - 2021
Prospective: 2021 - 2026
Standard therapy control cohort: Ages 4 - 65 years; receipt of disease-modifying therapy only between 2003 - 2026; followed as part of the VUMC-based Tennessee Never Lost Cohort.
EXCLUSION CRITERIA:
Participants judged to be non-compliant based on previous experience in terms of clinic appointments and following advice.
Principal Investigator
Referral Contact
For more information: