Unblinded and blinded N-of-1 trials versus usual care: a randomized controlled trial to increase statin uptake in primary care
<p><strong>Background:</strong> The aim was to assess whether an intervention incorporating a practicable open-label n-of-1 trial would lead to greater uptake of statin than usual care and comparable uptake to a closed-label gold-standard n-of-1 trial.</p> <p><stron...
| Main Authors: | , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
American Heart Association
2022
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| _version_ | 1826309068016320512 |
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| author | Tudor, K Brooks, J Howick, J Fox, R Aveyard, P |
| author_facet | Tudor, K Brooks, J Howick, J Fox, R Aveyard, P |
| author_sort | Tudor, K |
| collection | OXFORD |
| description | <p><strong>Background:</strong> The aim was to assess whether an intervention incorporating a practicable open-label n-of-1 trial would lead to greater uptake of statin than usual care and comparable uptake to a closed-label gold-standard n-of-1 trial.</p>
<p><strong>Methods:</strong> We enrolled patients who had stopped or declined statins into a 3-arm trial (usual care, unblinded, and blinded n-of-1 intervention arms). Physicians advised participants randomized to usual care to take statin therapy to prevent cardiovascular disease. In both intervention arms, physicians delivered a theoretically informed informed intervention endorsing the value of experimenting with medication in n-of-1 trials to assess whether it caused side-effects. In these trials, participants alternated between 4 weeks of medication and no medication (unblinded arm) or randomly sorted active and placebo (blinded arm) and recorded symptoms and symptom attributions for 6 months. Thereafter, physicians discussed participants’ symptom reports during active/inactive treatment periods and asked participants to resume statins if appropriate.</p>
<p><strong>Results:</strong> Seventy-three were randomized to the intervention arms and 20 to the control group. Fifty-six of 73 (77%) attempted the n-of-1 experiment; 28/36 (78%) in the unblinded arm; and 28/37 (76%) in the blinded arm. Forty-three of 56 (77%) completed the 6-month experiment and received feedback from the physician; 20/28 (71%) in the unblinded arm and 23/28 (82%) in the blinded arm. Thirty-three of 76 (45%) people restarted statins in the n-of-1 arms compared with 4/20 (20%) in the control arm, difference 24% (95% CI, 5%–43%; P=0.041). There was no evidence this differed between blinded and unblinded arms, difference 2% (95% CI, −20% to 24%; P=0.86). Adverse events occurred at a similar rate on and off statin.</p>
<p><strong>Conclusions:</strong> In patients refusing or intolerant of statin, supporting experimentation with n-of-1 trials increases medication uptake compared with usual care. Alternating on-off medication in unblinded n-of-1 experiments appears as effective as a blinded experiment.</p> |
| first_indexed | 2024-03-07T07:28:44Z |
| format | Journal article |
| id | oxford-uuid:56b9d281-5ea1-46d7-991d-8d77d4475871 |
| institution | University of Oxford |
| language | English |
| last_indexed | 2024-03-07T07:28:44Z |
| publishDate | 2022 |
| publisher | American Heart Association |
| record_format | dspace |
| spelling | oxford-uuid:56b9d281-5ea1-46d7-991d-8d77d44758712022-12-14T09:33:24ZUnblinded and blinded N-of-1 trials versus usual care: a randomized controlled trial to increase statin uptake in primary careJournal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:56b9d281-5ea1-46d7-991d-8d77d4475871EnglishSymplectic ElementsAmerican Heart Association2022Tudor, KBrooks, JHowick, JFox, RAveyard, P<p><strong>Background:</strong> The aim was to assess whether an intervention incorporating a practicable open-label n-of-1 trial would lead to greater uptake of statin than usual care and comparable uptake to a closed-label gold-standard n-of-1 trial.</p> <p><strong>Methods:</strong> We enrolled patients who had stopped or declined statins into a 3-arm trial (usual care, unblinded, and blinded n-of-1 intervention arms). Physicians advised participants randomized to usual care to take statin therapy to prevent cardiovascular disease. In both intervention arms, physicians delivered a theoretically informed informed intervention endorsing the value of experimenting with medication in n-of-1 trials to assess whether it caused side-effects. In these trials, participants alternated between 4 weeks of medication and no medication (unblinded arm) or randomly sorted active and placebo (blinded arm) and recorded symptoms and symptom attributions for 6 months. Thereafter, physicians discussed participants’ symptom reports during active/inactive treatment periods and asked participants to resume statins if appropriate.</p> <p><strong>Results:</strong> Seventy-three were randomized to the intervention arms and 20 to the control group. Fifty-six of 73 (77%) attempted the n-of-1 experiment; 28/36 (78%) in the unblinded arm; and 28/37 (76%) in the blinded arm. Forty-three of 56 (77%) completed the 6-month experiment and received feedback from the physician; 20/28 (71%) in the unblinded arm and 23/28 (82%) in the blinded arm. Thirty-three of 76 (45%) people restarted statins in the n-of-1 arms compared with 4/20 (20%) in the control arm, difference 24% (95% CI, 5%–43%; P=0.041). There was no evidence this differed between blinded and unblinded arms, difference 2% (95% CI, −20% to 24%; P=0.86). Adverse events occurred at a similar rate on and off statin.</p> <p><strong>Conclusions:</strong> In patients refusing or intolerant of statin, supporting experimentation with n-of-1 trials increases medication uptake compared with usual care. Alternating on-off medication in unblinded n-of-1 experiments appears as effective as a blinded experiment.</p> |
| spellingShingle | Tudor, K Brooks, J Howick, J Fox, R Aveyard, P Unblinded and blinded N-of-1 trials versus usual care: a randomized controlled trial to increase statin uptake in primary care |
| title | Unblinded and blinded N-of-1 trials versus usual care: a randomized controlled trial to increase statin uptake in primary care |
| title_full | Unblinded and blinded N-of-1 trials versus usual care: a randomized controlled trial to increase statin uptake in primary care |
| title_fullStr | Unblinded and blinded N-of-1 trials versus usual care: a randomized controlled trial to increase statin uptake in primary care |
| title_full_unstemmed | Unblinded and blinded N-of-1 trials versus usual care: a randomized controlled trial to increase statin uptake in primary care |
| title_short | Unblinded and blinded N-of-1 trials versus usual care: a randomized controlled trial to increase statin uptake in primary care |
| title_sort | unblinded and blinded n of 1 trials versus usual care a randomized controlled trial to increase statin uptake in primary care |
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