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Prevention of Morbidity in Sickle Cell Disease (POMS2a)-overnight auto-adjusting continuous positive airway pressure compared with nocturnal oxygen therapy: a randomised crossover pilot study examining patient preference and safety in adults and children

Prevention of Morbidity in Sickle Cell Disease (POMS2a)-overnight auto-adjusting continuous positive airway pressure compared with nocturnal oxygen therapy: a randomised crossover pilot study examining patient preference and safety in adults and children
Prevention of Morbidity in Sickle Cell Disease (POMS2a)-overnight auto-adjusting continuous positive airway pressure compared with nocturnal oxygen therapy: a randomised crossover pilot study examining patient preference and safety in adults and children

DESIGN: This randomised crossover trial compared nocturnal auto-adjusting continuous positive airway pressure (APAP) and nocturnal oxygen therapy (NOT) in adults and children with sickle cell anaemia, with patient acceptability as the primary outcome. Secondary outcomes included pulmonary physiology (adults), safety, and daily pain during interventions and washout documented using tablet technology.

METHODS: Inclusion criteria were age > 8 years and the ability to use an iPad to collect daily pain data. Trial participation was 4 weeks; week 1 involved baseline data collection and week 3 was a washout between interventions, which were administered for 7 days each during weeks 2 and 4 in a randomised order. Qualitative interviews were transcribed verbatim and analysed for content using a funnelling technique, starting generally and then gaining more detailed information on the experience of both interventions. Safety data included routine haematology and median pain days between each period. Missing pain day values were replaced using multiple imputation.

RESULTS: Ten adults (three female, median age 30.2 years, range 18-51.5 years) and eleven children (five female, median age 12 years, range 8.7-16.9 years) enrolled. Nine adults and seven children completed interviews. Qualitative data revealed that the APAP machine was smaller, easier to handle, and less noisy. Of 16 participants, 10 preferred APAP (62.5%, 95% confidence interval (CI) 38.6-81.5%). Haemoglobin decreased from baseline on APAP and NOT (mean difference -3.2 g/L (95% CI -6.0 to -0.2 g/L) and -2.5 g/L (95% CI -4.6 to 0.3 g/L), respectively), but there was no significant difference between interventions (NOT versus APAP, 1.1 (-1.2 to 3.6)). Pulmonary function changed little. Compared with baseline, there were significant decreases in the median number of pain days (1.58 for APAP and 1.71 for NOT) but no significant difference comparing washout with baseline. After adjustment for carry-over and period effects, there was a non-significant median difference of 0.143 (95% CI -0.116 to 0.401) days additional pain with APAP compared with NOT.

CONCLUSION: In view of the point estimate of patient preference for APAP, and no difference in haematology or pulmonary function or evidence that pain was worse during or in washout after APAP, it was decided to proceed with a Phase II trial of 6 months APAP versus standard care with further safety monitoring for bone marrow suppression and pain.

TRIAL REGISTRATION: ISRCTN46078697 . Registered on 18 July 2014.

1745-6215
1-15
Howard, Jo
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Lee, Sophie A
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Inusa, Baba
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Cheng, Man Ying Edith
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Bavenjit, Cheema
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Reading, Isabel C.
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Wakeford, Sally Ann
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Gavlak, Johanna C
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Murphy, Patrick B
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Hart, Nicholas
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Gupta, Atul
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Sahota, Sati
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Jacob, Eufemia
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Chorozoglou, Maria
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Ossai, Carol
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Gwam, Maureen
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Kirkham, Fenella J.
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Wade, Angela M
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Liossi, Christina
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Howard, Jo
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Lee, Sophie A
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Inusa, Baba
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Cheng, Man Ying Edith
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Bavenjit, Cheema
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Reading, Isabel C.
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Wakeford, Sally Ann
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Gavlak, Johanna C
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Murphy, Patrick B
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Hart, Nicholas
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Gupta, Atul
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Sahota, Sati
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Jacob, Eufemia
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Chorozoglou, Maria
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Ossai, Carol
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Gwam, Maureen
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Kirkham, Fenella J.
1dfbc0d5-aebe-4439-9fb2-dac6503bcd58
Wade, Angela M
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Liossi, Christina
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Howard, Jo, Lee, Sophie A, Inusa, Baba, Cheng, Man Ying Edith, Bavenjit, Cheema, Reading, Isabel C., Wakeford, Sally Ann, Gavlak, Johanna C, Murphy, Patrick B, Hart, Nicholas, Gupta, Atul, Sahota, Sati, Jacob, Eufemia, Chorozoglou, Maria, Ossai, Carol, Gwam, Maureen, Kirkham, Fenella J., Wade, Angela M and Liossi, Christina (2019) Prevention of Morbidity in Sickle Cell Disease (POMS2a)-overnight auto-adjusting continuous positive airway pressure compared with nocturnal oxygen therapy: a randomised crossover pilot study examining patient preference and safety in adults and children. Trials, 20, 1-15, [442]. (doi:10.1186/s13063-019-3461-x).

Record type: Article

Abstract

DESIGN: This randomised crossover trial compared nocturnal auto-adjusting continuous positive airway pressure (APAP) and nocturnal oxygen therapy (NOT) in adults and children with sickle cell anaemia, with patient acceptability as the primary outcome. Secondary outcomes included pulmonary physiology (adults), safety, and daily pain during interventions and washout documented using tablet technology.

METHODS: Inclusion criteria were age > 8 years and the ability to use an iPad to collect daily pain data. Trial participation was 4 weeks; week 1 involved baseline data collection and week 3 was a washout between interventions, which were administered for 7 days each during weeks 2 and 4 in a randomised order. Qualitative interviews were transcribed verbatim and analysed for content using a funnelling technique, starting generally and then gaining more detailed information on the experience of both interventions. Safety data included routine haematology and median pain days between each period. Missing pain day values were replaced using multiple imputation.

RESULTS: Ten adults (three female, median age 30.2 years, range 18-51.5 years) and eleven children (five female, median age 12 years, range 8.7-16.9 years) enrolled. Nine adults and seven children completed interviews. Qualitative data revealed that the APAP machine was smaller, easier to handle, and less noisy. Of 16 participants, 10 preferred APAP (62.5%, 95% confidence interval (CI) 38.6-81.5%). Haemoglobin decreased from baseline on APAP and NOT (mean difference -3.2 g/L (95% CI -6.0 to -0.2 g/L) and -2.5 g/L (95% CI -4.6 to 0.3 g/L), respectively), but there was no significant difference between interventions (NOT versus APAP, 1.1 (-1.2 to 3.6)). Pulmonary function changed little. Compared with baseline, there were significant decreases in the median number of pain days (1.58 for APAP and 1.71 for NOT) but no significant difference comparing washout with baseline. After adjustment for carry-over and period effects, there was a non-significant median difference of 0.143 (95% CI -0.116 to 0.401) days additional pain with APAP compared with NOT.

CONCLUSION: In view of the point estimate of patient preference for APAP, and no difference in haematology or pulmonary function or evidence that pain was worse during or in washout after APAP, it was decided to proceed with a Phase II trial of 6 months APAP versus standard care with further safety monitoring for bone marrow suppression and pain.

TRIAL REGISTRATION: ISRCTN46078697 . Registered on 18 July 2014.

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Accepted/In Press date: 20 May 2019
Published date: 18 July 2019

Identifiers

Local EPrints ID: 433395
URI: http://eprints.soton.ac.uk/id/eprint/433395
ISSN: 1745-6215
PURE UUID: 753e5480-f1c4-4040-bbae-85267add839d
ORCID for Isabel C. Reading: ORCID iD orcid.org/0000-0002-1457-6532
ORCID for Fenella J. Kirkham: ORCID iD orcid.org/0000-0002-2443-7958
ORCID for Christina Liossi: ORCID iD orcid.org/0000-0003-0627-6377

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Date deposited: 19 Aug 2019 16:30
Last modified: 07 Oct 2020 06:10

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Contributors

Author: Jo Howard
Author: Sophie A Lee
Author: Baba Inusa
Author: Man Ying Edith Cheng
Author: Cheema Bavenjit
Author: Isabel C. Reading ORCID iD
Author: Sally Ann Wakeford
Author: Johanna C Gavlak
Author: Patrick B Murphy
Author: Nicholas Hart
Author: Atul Gupta
Author: Sati Sahota
Author: Eufemia Jacob
Author: Carol Ossai
Author: Maureen Gwam
Author: Fenella J. Kirkham ORCID iD
Author: Angela M Wade

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