Sickle Cell Disease
Conditions
Keywords
NIS, sickle cell disease, SCD, crizanlizumab
Brief summary
This is a multicenter, prospective, single-arm observational non-interventional study (NIS), which will be conducted in various countries in the Middle East and India.
Detailed description
The study is designed to collect information on the utilization and effectiveness of crizanlizumab treatment in SCD patients under routine clinical practice conditions, to which the physician has made an independent decision to prescribe crizanlizumab.
Interventions
OTHERCrizanlizumab
Prospective observational study. There is no treatment allocation. Patients administered crizanlizumab, that have started before inclusion of the patient into the study will be enrolled.
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
16 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
1. Male and female patients with clinical diagnosis of SCD (based on laboratory parameters) of any genotype. 2. Patients newly initiated on treatment with locally approved crizanlizumab. 3. Patients aged 16 years or older at crizanlizumab initiation.
Exclusion criteria
1. Patients who did not provide informed consent. 2. Patients who received a stem cell transplant at time of enrollment. 3. Patients who participated in or are participating in a clinical trial at time of enrollment or in the 12 months prior to starting commercial crizanlizumab. 4. According to the investigator's opinion, the patient is an unlikely candidate to provide an accurate medical history and/or to obtain long-term follow-up information for any reasons such as unavailability or severe concomitant illnesses.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualized rate of healthcare visit and home-managed (VOCs) requiring a medical facility visit | 12 months | The annualized rate of VOCs is defined as the total number of pain crises for a patient occurring from the date of initial infusion with crizanlizumab (commercially available) to the last contact date of the study end date × 365 divided by the number of days during that same time period. This calculation accounts for early dropouts or lost to follow-up by extrapolating the VOC rate of every patient to 1 year. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Dose of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home. | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Dose of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home to be collected |
| Duration of use of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home. | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Duration of use of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home to be collected |
| Number of healthcare visit and home-managed (VOCs) leading to hospitalization | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Number of healthcare visit and home-managed (VOCs) leading to hospitalization to be collected |
| Type of healthcare visit and home-managed (VOCs) leading to hospitalization | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Type of healthcare visit and home-managed (VOCs) leading to hospitalization to be collected |
| Duration of healthcare visit and home-managed (VOCs) leading to hospitalization | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Duration of healthcare visit and home-managed (VOCs) leading to hospitalization to be collected |
| Outcome of healthcare visit and home-managed (VOCs) leading to hospitalization | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Outcome of healthcare visit and home-managed (VOCs) leading to hospitalization to be collected. |
| Type of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home. | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Type of opioids for pain management of healthcare visit and home-managed (VOCs) within the medical facility and at home to be collected |
| Annualized rate home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S. | month 12 and month 24 | Annualized rate home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S to be collected. SCPD-S: Sickle Cell Pain Diary - Self Report |
| Percentage of patients free from healthcare visit and home-managed (VOCs) leading to a healthcare visit | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Percentage of patients free from healthcare visit and home-managed (VOCs) leading to a healthcare visit to be collected |
| Percentage of patients with acute and chronic complications/ end organ damage related to SCD | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Percentage of patients with acute and chronic complications/ end organ damage related to SCD to be collected |
| Frequency of patients on blood transfusions | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Frequency of patients on blood transfusions to be collected. Blood transfusions: simple/ exchange/ chronic/ episodic |
| Frequency of patients with SCD-related Healthcare Resource Utilization (HRU) | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Frequency of patients with SCD-related Healthcare Resource Utilization (HRU) to be collected |
| Number of patients with clinical laboratory parameters abnormalities | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Number of patients with clinical laboratory parameters abnormalities to be collected |
| Number of home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S. | Baseline, 3 months, 6 months, 12 months, 18 months, and 24 months | Number of home managed healthcare visit and home-managed (VOCs) as captured in the medical records and as per data from the SCPD-S to be collected. SCPD-S: Sickle Cell Pain Diary - Self Report |
Countries
Bahrain, Kuwait, Qatar, Saudi Arabia
Outcome results
None listed