APPENDICES - NIHR Health Technology Assessment Programme

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Appendix 15
Study details Population details Treatment details Results Interpretation
Authors’ conclusions This
study demonstrated that lesion
recurrence rate with MAL–PDT
treatment was comparable to
double freeze thaw cryotherapy
for treatment of superficial BCC
and provided a better cosmetic
outcome
Brief study appraisal Overall
this trial was well conducted
clearly reported. The lack of
a power calculation means
it is unclear if there was
no difference between the
treatments, or if the study was
underpowered to detect such a
difference. This trial did include
a long term FU of 5 yr as well
as examination of safety, efficacy
and cosmetic outcomes
Morbidity
3 mth (115 patients): Lesions with inCR after 3 mth were 32% in
the PDT group and 30% in the cryotherapy group. CR rates did
not differ between the groups (PDT: 97% vs cryotherapy: 95%,
p = 0.49)
12 mth (105 patients): Fewer lesions recurred with MAL–PDT
than with cryotherapy (8% vs 16%) More patients had an
‘excellent/good’ cosmetic outcome with MAL–PDT than with
cryotherapy at 3 and 12 mth
36 mth (107 patients): Proportion of lesions in CR was 66% for
MAL–PDT and 67% for cryotherapy (NS). 74% estimated CR
rate in both groups according to per-protocol population. The
lesion recurrence rates in lesions with CR 3 mth after the last
treatment were 23% for MAL–PDT and 20% for cryotherapy
The overall cosmetic outcome was rated by physicians as
‘excellent’ or ‘good’ for 89% of the MAL–PDT patients and 63%
of the cryotherapy patients
5 yr: CR rates did not differ between the groups (PDT: 75% vs
cryotherapy: 74%, p = 0.90). Cumulative recurrence rate after 5
yr was PDT: 22% and cryotherapy: 20%, p = 0.86
QoL and return to normal activity Cosmetic outcome
was better with PDT at both 3 mth and 5 yr 3 mth: 30% of PDT
patients had an ‘excellent’ outcome compared with 4% for
cryotherapy (p = 0.0005)
5 yr: 60% of PDT patients had an ‘excellent’ outcome compared
with 16% for cryotherapy (p = 0.00078)
All cosmetic outcomes rated by investigators using a 4-point
scale
AEs AEs were reported by 73% (44/60) PDT patients and 79%
(46/58) cryotherapy patients. Most AEs were local and transient,
no patients discontinued as a result of treatment-related AEs
Pain: 37% PDT, 33% cryotherapy
Crusting: 35% PDT, 47% cryotherapy
Erythema: 30% PDT, 21% cryotherapy
Mild: 80% PDT 73% cryotherapy
Moderate: 13% PDT, 25% cryotherapy
Severe: 5% PDT, 1% cryotherapy
Trial treatments MAL–PDT
vs Cryotherapy
Intervention MAL–PDT: A
single treatment was initially
given. Lesions were prepared
by surface debridement.
MAL cream, 160 mg/g, was
applied in a layer of 1 mm
to the lesion and 5 mm of
surrounding tissue for 3 hr. The
cream was washed off using a
saline solution and the treated
area was then illuminated
with non-coherent red light
(wavelength 570–670 nm)
using a light dose of 75 J/cm.
In patients with incomplete
CR at 3-mth treatment was
repeated (two consecutive
MAL–PDT sessions 7 d apart)
Comparator Cryotherapy:
Cryotherapy was applied
in two freeze–thaw cycles
using liquid nitrogen spray
applied to the lesion and a
3-mm surrounding area of
healthy tissue. Procedure was
repeated after a thaw period
of two to three times the
freeze duration. In patients
with an incomplete response
at 3-mth treatment was
repeated (double freeze–thaw
cryotherapy)
Treatment intention
Curative
Type(s) of cancer
and histology Primary
superficial BCC
Main eligibility
criteria Patients aged
18 yr or older with
up to 10 previously
untreated primary
superficial BCC
lesions suitable for
cryotherapy. Diagnosis
confirmed using punch
biopsy. Lesions had to
have diameter > 6 mm
but < 15 mm on face/
scalp, < 20 mm on neck/
extremities, or < 30 mm
on trunk. Further
eligibility criteria were
reported
Patient
characteristics Not
stated
Concomitant
treatment
Concomitant
treatment with
immunosuppressive
medication was
prohibited
Authors Bassett-
Seguin et al. (2008) 79
Linked
publications186–190 Data source Full
published paper
Country Not stated
Language English
Study design RCT
(between-participant
comparison)
No. of participants
Total: 120 randomised,
118 treated (219
lesions)
Intervention: 60, 58
treated and analysed
(103 lesions)
Comparator: 58, 57
treated and analysed
(98 lesions)
No. of recruiting
centres 13 across
seven European
countries
Follow-up period
and frequency 3
mth, then at 1, 2, 3, 4
and 5 yr