Prevention of Morbidity in sickle cell disease--qualitative outcomes, pain and quality of life in a randomised cross-over pilot trial of overnight supplementary oxygen and auto-adjusting continuous positive airways pressure (POMS2a): study protocol for a randomised controlled trial

• Published in: Current controlled trials in cardiovascular medicine 2015-08, Vol.16 (1), p.376-376, Article 376
• Main Authors: Howard, Jo, Inusa, Baba, Liossi, Christina, Jacob, Eufemia, Murphy, Patrick B, Hart, Nicholas, Gavlak, Johanna, Sahota, Sati, Chorozoglou, Maria, Nwosu, Carol, Gwam, Maureen, Gupta, Atul, Rees, David C, Thein, Swee Lay, Reading, Isabel C, Kirkham, Fenella J, Cheng, Man Yeung Edith
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Airway management; Analysis; Anemia, Sickle Cell - complications; Anemia, Sickle Cell - diagnosis; Anemia, Sickle Cell - physiopathology; Anemia, Sickle Cell - psychology; Care and treatment; Child; Clinical Protocols; Clinical trials; Complications and side effects; Continuous Positive Airway Pressure - adverse effects; Continuous Positive Airway Pressure - methods; Cross-Over Studies; Disease prevention; England; Female; Hematology; Hemoglobin; Hospitals; Humans; Hypoxia; Intervention; Interviews as Topic; Lung diseases; Male; Morbidity; Oximetry; Oxygen Inhalation Therapy - adverse effects; Oxygen saturation; Oxygen therapy; Pain; Patient Satisfaction; Pediatrics; Physiology; Pilot Projects; Prevention; Quality of Life; Research Design; Respiratory Function Tests; Risk factors; Sickle cell anemia; Sickle cell disease; Sleep Apnea Syndromes - diagnosis; Sleep Apnea Syndromes - etiology; Sleep Apnea Syndromes - physiopathology; Sleep Apnea Syndromes - psychology; Sleep Apnea Syndromes - therapy; Sleep disorders; Smartphones; Study Protocol; Surveys and Questionnaires; Time Factors; Treatment Outcome; Young Adult
• ISSN: 1745-6215; 1745-6215
• DOI: 10.1186/s13063-015-0883-y

Sickle cell anaemia (SCA) is an inherited disorder of haemoglobin. Patients experience long-term health care problems, affecting quality of life (QOL) including frequent acute pain, which is difficult to document in trials except as hospital admissions. Pilot data suggests that overnight respiratory support, either supplementary oxygen or auto-adjusting continuous positive airways pressure (APAP), is safe and may have clinical benefit. This pilot trial aims to determine which intervention is more acceptable to participants and whether there are other advantages of one over the other, e.g. in respiratory function or haematological parameters, before conducting the Phase 2 trial of overnight respiratory support funded by the National Institutes of Health Research. This is a pilot cross-over interventional trial with the order of interventions decided by simple randomization. Ten adults (age over 18 years) and 10 children (aged between 8 and 18 years) with homozygous sickle cell disease (haemoglobin SS, HbSS), recruited regardless of symptoms of sleep-disordered breathing, will undergo overnight pulse oximetry and will have two interventions, overnight oxygen and APAP, for a week each in randomised order with a washout week between interventions. Participants will complete online diaries via an iPad throughout the 29 days of the study and will complete QOL questionnaires and have measurement of haematology, biochemistry, spirometry and lung volumes (adults only) at 3 time points, at baseline and after each intervention, as well as in-depth semi-structured qualitative interviews after each intervention, carried out by an experienced psychologist. Both qualitative and statistical methods will be used to analyze the data. The primary outcome is qualitative data looking at participant experience from the transcribed interviews after each intervention. The participant's view on feasibility, acceptability and preference will specifically be explored. The QOL, laboratory and lung function data will be compared with baseline for each arm. Patient and public involvement is an integral part of this trial and the key outcome is the qualitative result, which is dependent on obtaining good quality data to advise on participant feasibility, acceptability and preference. This is being addressed by using a standard interview. The development of a pain endpoint is another important outcome and collecting daily measurements is likely to be challenging. Research results will be