A Study to Learn About Sickle Cell Disease In Adult Patients

Participants with a confirmed diagnosis of stable Sickle Cell Disease (SCD) (hemoglobin S inherited from both parents [HbS/S] or hemoglobin S inherited from one parent and hemoglobin beta thalassemia inherited from the other parent [HbS/beta-zero-thalassemia] genotype) who are either not on disease modifying treatment or on a stable dose of a SCD disease modifying treatment regimen.

Inclusion Criteria (All Groups):

- Confirmed diagnosis of stable SCD (HbS/S or HbS/beta-zero-thalassemia).

Additional Inclusion Criteria (No Disease Modifying Treatment Control Group):

• Have experienced ≥1 episode(s) of medical utilization (MU) VOC within 12 months prior to Screening.
• Data available for number of MU VOC(s) during the 12-month interval prior to Screening and a value for %fetal hemoglobin (HbF) collected subsequent to 1 year of age in the absence of recent transfusion.

Additional Inclusion Criteria (SCD Disease Modifying Treatment Group):

• Have experienced ≥1 episode(s) of MU VOC within 12 months prior to initiation of HU and/or crizanlizumab (whichever was initiated earlier).

• Must be on a stable dose of their SCD treatment regimen ≥8 weeks prior to Day 1 with the intent of remaining on the same dose throughout the study, unless adjustments are medically necessary due to bone marrow suppression, in accordance with published guidelines and/or product specific guidance (eg, package label). Accepted SCD disease modifying treatment regimens include:

  • HU alone and/or in combination with crizanlizumab, L-glutamine and/or voxelotor; or
  • Crizanlizumab alone and/or in combination with HU, L-glutamine and/or voxelotor.
• Data available for number of MU VOC(s) during the 12-month interval prior to initiation of any SCD disease modifying treatment, as described above, and a value for %HbF collected subsequent to 1 year of age, prior to initiation of any HU treatment, and in the absence of recent transfusion.

Exclusion Criteria (All Groups):

• Evidence or history of ongoing (condition or sequelae) clinically significant hematological (non-SCD), renal, endocrine, pulmonary, gastrointestinal, cardiovascular (including overt stroke but excluding silent cerebral infarct), hepatic (excluding cholelithiasis), psychiatric or neurological disease as assessed from medical records.
• Marked ongoing bone marrow suppression as evidenced by any of the following as per medical record: severe anemia, absolute neutrophil count (ANC) <1000 mm3 white blood cell (WBC), thrombocytopenia (platelet count <100,000 mm3) within ≤8 weeks prior to Day 1 enrollment.
• History of hematopoietic stem cell transplant or treatment with gene therapy as assessed from medical records.
• History of simple transfusion within ≤4 weeks prior to Day 1 enrollment as assessed from medical records or participant self-report.
• History of chronic transfusion/exchange transfusion within ≤12 weeks prior to Day 1 enrollment as assessed from medical records or participant self-report and/or plan to initiate such treatment during the 6-month observation period.

Additional Exclusion Criteria (No Disease Modifying Treatment Control Group):

• Participant received HU and/or crizanlizumab at any time within ≤18 months of Day 1 enrollment and treatment(s) was discontinued due to lack of efficacy (no reduction in the frequency of VOCs, documented or perceived) and/or plan to initiate said treatment(s) during the 6-month observation period.
• Participant received voxelotor or L-glutamine within ≤4 weeks of Day 1 enrollment and/or plan to initiate said treatment(s) during the 6-month observation period.

Pfizer

Vaso-occlusive crisis (VOC) Hemoglobin S (HbS)

Layout table for MeSH terms

Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia	Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn