A patient-initiated treatment model for blepharospasm and hemifacial spasm: a randomized controlled trial

Background: To test, in a two-arm, single center, superiority, randomized controlled trial, the efectiveness of and
costs associated with a patient-initiated treatment model for people with hemifacial spasm (HFS) and blepharospasm
(BEB) in comparison to usual care.
Methods: One hundred and thirty patients with HFS or BEB, aged 18 years or over, were recruited from a nurse-led
botulinum toxin type A clinic at an eye hospital in the United Kingdom (UK), completed baseline measures and were
randomized (1:1). The intervention group determined their own botulinum toxin type A (BoNT/A) treatment schedule
during the trial period (9months) and received an information leafet with a “hotline” number to book an appoint‑
ment. Usual care appointments were scheduled by treating clinicians. Data analysts were blind to study group. The
primary outcomes were disease severity and functional disability, as measured by the Jankovic Rating Scale and
Blepharospasm Disability Index, respectively. Secondary outcomes included quality of life, anxiety and depression,
satisfaction with care, confdence in the service, economic costs and employment days lost.
Results: Sixty-fve patients were randomized to each group. The intervention demonstrated no statistically signif‑
cant diference to usual care for any of primary outcomes. On secondary outcomes the levels of anxiety difered
signifcantly (F2, 142.39 =1.65, p =0.02), with the intervention arm exhibiting a decrease and the control arm an
increase (Hedges’ g=−0.26 [99% CI -0.83, 0.32]). No other statistically signifcant diferences were found for sec‑
ondary outcomes. Overall healthcare costs and costs to the patient were on average £198.95 less (95% CI -£256.76,
£654.67; p =0.10) per participant for those in the intervention compared to usual care, although this fnding was not
signifcant.
Conclusions: We did not observe diferences between the patient-initiated treatment model and usual care for peo‑
ple with BEB or HFS, on any primary outcome measure, quality of life, or depression. The patient-initiated treatment
model may, however, have the potential to save healthcare costs and reduce anxiety. Patients using this new model
were also equally as satisfed in the service and confdent in their care as those receiving treatment as usual.