A Retrospective Data Collection and Analysis Study of Patients With Sickle Cell Disease (SCD) Who Have Been Treated With Oxbryta® (Voxelotor)

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Case-control

Study drug and medical condition

• Name of medicine:
  OXBRYTA
• Study drug International non-proprietary name (INN) or common name:
  VOXELOTOR
• Anatomical Therapeutic Chemical (ATC) code:
  (B06AX03) voxelotor

  voxelotor
• Medical condition to be studied:
  Sickle cell disease

Population studied

• Short description of the study population:
  1. Willing and able to provide written informed consent (ages >= 18 years) or parental/guardian consent and patient assent (age <18 years), as required by the IRB, institution, or per local regulations
• Age groups:
  Paediatric Population (< 18 years)

  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  300

Study design details

• Study design:
  This is a multicenter, retrospective data collection and analysis study to characterize health outcomes in approximately 300 patients with SCD who have been treated with Oxbryta as part of their usual care. Data will be collected at approximately 10 sites in the US.
• Main study objective:
  Primary; The following are categories of interest in patients with SCD treated with Oxbryta:; • Clinical outcomes, as assessed by clinical and laboratory assessments of hematological parameters and end organ damage, and incidence of significant clinical events; • Healthcare resource utilization; • Health-related quality of life (HRQoL), as assessed by patient-reported outcome (PRO) measures and clinician-reported outcomes (ClinRO); ; Safety; The safety objective is to assess the safety and tolerability of Oxbryta.
• Setting:
  This is a multicenter, retrospective data collection and analysis study to characterize health outcomes in approximately 300 patients with SCD who have been treated with Oxbryta as part of their usual care.; Any patient with SCD who received Oxbryta treatment for at least 2 weeks as part of their usual care according to the Oxbryta US Prescribing Information (USPI) is eligible to participate.; Patients will be introduced to the study by their health care team and will sign the informed consent form (ICF) to allow their data to be collected and used for the study, if required by the Institutional Review Board (IRB), institution, or per local regulations.; Only data that are available from the patient's medical records and other secondary data sources will be collected. Study data from 1 year before and up to 1 year after the first dose of Oxbryta will be entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff.
• Outcomes:
  • Change from pre-Oxbryta treatment period in the following hematologic parameters corresponding to treatment with Oxbryta:; o Hb; o Hemolysis measures, including % reticulocytes, absolute reticulocytes, bilirubin (total, direct, and indirect); o Measures of iron overload, including ferritin, iron, total iron binding capacity (TIBC), T2-weighted magnetic resonance imaging (T2*MRI), and liver biopsy; • Change from pre-Oxbryta treatment period in renal function, as measured by the following:; o Albuminuria (urine albumin/creatinine ratio [ACR]); o Hemoglobinuria (urine dipstick positive for blood +1 or greater and :: 2 RBC by high power field); o Serum cystatin C; o Estimated glomerular filtration rat (eGFR) calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation; • Incidence of significant SCD-related clinical events, such as vaso-occlusive crisis (VOC), acute chest syndrome (ACS), priapism, cerebral infarcts, transient ischemic attack (TIA), leg ulcers, and measures of cardiac function and pulmonary hypertension (PH); • Treatment initiation or modification of SCD-related medications; (e.g., hydroxyurea, crizanlizumab, L-glutamine, opioids [in daily morphine equivalents], iron chelating agents, erythropoiesis-stimulating agents [ESAs], nonsteroidal anti-inflammatory drugs [NSAIDs], folic acid, and penicillin); • Change from pre-Oxbryta treatment period in healthcare resource utilization: incidence of unplanned clinic visits, emergency department (ED) visits, hospitalizations (including total length of stay and time in intensive care unit [ICU], if applicable), acute and chronic RBC transfusions, home oxygen supplementation, and renal dialysis; • Change from pre-Oxbryta treatment period in the following HRQoL measures:; o Patient-Reported Outcomes Measurement Information System (PROMIS); o Patient Global Impression of Change (PGIC); o Clinical Global Impression of Change (CGIC);
• Data analysis plan:
  After informed consent/assent has been obtained (if required by the institution or IRB), the data listed below that are available at or around the timepoints indicated in the schedule of activities (SOA) in Appendix 1 will be collected and entered into the EDC by study personnel at the study site. Data collection will include only those data that are recorded in the patient's medical records and other secondary data sources, up to the timepoint when the data is extracted. Study data that are not available in medical records or other secondary data sources will not be solicited from patients.