Sickle cell disease Haematological Disorders Sickle-cell disorders
Conditions
Interventions
Patients will be randomised to six months of overnight APAP treatment or to standard care alone. Patients will not be blinded to treatment as the use of a sham treatment was not thought to be ethical.
Sponsors
University Hospital Southampton (UK)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age >=8 years 2. Informed consent with assent in accordance with the institutional policies (UK ethical committee) and European guidelines must be signed by the patient or patient's parent or legally authorised guardian acknowledging written consent to join the study. Patients < 16 years will be requested to give their assent to join the study 3. HbSS diagnosed by standard techniques (HPLC, IEF and MS). Participating institutions must submit documentation of the diagnostic haemoglobin analysis 4. Able to speak and understand English 5. Patient or parent/guardian able to use smartphone app 6. Overnight oximetry showing minimum overnight oxygen saturation of <94%
Exclusion criteria
Exclusion criteria: 1. Patient already on overnight respiratory support, or has used it in the past 2. Hospital admission for acute sickle complication within the past 1 month 3. Patient with >6 admissions for acute sickle complications within the past 12 months 4. Existing respiratory failure 5. Overnight oximetry showing mean overnight saturation of 30% of total measured time 6. Severe sleep apnoea (OSA) defined by 4% ODI > 15/hr; Epworth sleepiness score >10 7. Exclusions to APAP therapy: 7.1. Decompensated cardiac failure 7.2. History of severe epistaxis 7.3. Trans-sphenoidal surgery, or trauma that could have left a cranio-nasopharyngeal fistula 7.4. Perforated ear drum 8. Bullous lung disease 9. Bypassed upper airway 10. Pneumothorax 11. Patient at increased risk of aspiration 12 Pneumocephalus has been reported in a patient using nasal Continuous Positive Airway Pressure. Caution should be used when prescribing APAP for susceptible patients such as those with: cerebral spinal fluid (CSF) leaks, abnormalities of the cribriform plate, prior history of head trauma, and/or pneumocephalus 13. Pregnancy 14. Patients on chronic blood transfusion regimes, or has had blood transfusion within past 3 months 15. Any acute or chronic condition which would limit the patient’s ability to complete the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Pain is the most common symptoms experienced by patients with SCD and will be measured before starting treatment and in the last month of treatment using an electronic pain diary. This was used effectively in the first part of the study 2. Brain function will be measured before and after six months of treatment by a psychologist who will use several different tests. The trial will also look at MRI brain scans before and after treatment and at heart function Added 01/08/2017: As described in the study protocol (24/12/2015), this trial was powered on change in cancellation from the Wechsler scales comparing results before and after the intervention. | — |
Secondary
| Measure | Time frame |
|---|---|
| Current secondary outcome measures as of 01/08/2017: Based on the final study protocol (24/12/2015), the secondary outcomes of interest are: 1. Other Cognitive endpoints at baseline and after interventions 1.1. Change in assessment of processing speed/attention indices 1.2. Learning/memory indices and executive functions 2. Pain endpoints and pain characteristics/descriptors : 2.1. Current pain, using a 0 to 10 electronic numerical rating scale (e-NRS) 2.2. The highest e-NRS in past 24h 2.3. The lowest e-NRS in past 24h 2.4. The average-e-NRS in the past 24h 2.5. Location of pain on a body outline diagram 2.6. How much pain interfered with everyday activities in past 24h (2 week baseline compared with 2 week diary at end of study) The same measurements are obtained for one week at the end of the 2nd and 4th months after baseline. In addition at the end of every week a phone call and the above pain end points are recorded. The same measurements are obtained at the end of the two measurement weeks at the end of the 2nd and 4th months after baseline. 3. Quality of Life is measured via Peds-QL and EQ-5D/CHU-9D at baseline and six months 4. Physiological investigations 4.1. Daytime oximetry 4.2. Renal function 4.3. 6 minute walk 4.4. Echocardiography 4.5. MRI brain and heart 4.6. Biomarkers via blood spot 5. Safety endpoints: full blood count, reticulocytes at baseline, 2 weeks, 3 months and 6 months 6. Adverse events/Side effect profile are measured using adverse events and serious adverse events reported monthly 7. Health economic analysis using EQ-5D and client service receipt inventory (CSIR) inventory tha | — |
Countries
United Kingdom
Outcome results
None listed