Table 5.

Summary of category 1 and 2 cyclosporine A studies included in the SLR

Study	Study type	Baseline severity	Sample size (completers)	Treatments	Duration of disease	Response assessment(s)†	Response assessment period	Main efficacy findings	Tolerability/safety
Category 1: PBO-controlled RCT
Lai et al. [36]	PBO-controlled RCT	Moderate-to-severe AA**, mean SALT 79.4	N = 36 (N = 32)	CsA (4 mg/kg/d) N = 18 PBO N = 18	Duration of current episode: mean 6.5 Yr	Primary: SALT50 Pt achieving ≥ 1-grade improvement in EB/EL assessment scale score (0, none to 3, normal)	3 Mth	SALT50 CsA: 5/16 (31%) PBO: 1/16 (6%)  ≥ 1-grade improvement in EB score CsA: 5/16 (31%) PBO: 0/16 (0%) P = 0.02  ≥ 1-grade improvement in EL score CsA: 3/16 (19%) PBO: 0/16 (0%)	AE rates: CsA: 15/18 (83%) PBO: 15/18 (83%) AEs in ≥ 5% pt: headache, paresthesia, abdominal pain, nausea, UTI, musculoskeletal disorders, respiratory disorders, pruritis, hirsutism
Category 2: non-randomized trials, observational studies, RCT without PBO
Ferrando and Grimalt [37]	Non-randomized	Severe AA‡ AU, AT, patchy	N = 15	CsA (average: 150 mg BID)	Mean 9.8 Yr (range 1–24)	% hair regrowth	1–12 Mth	≥ 90% hair regrowth: 2/15 (13%)  ≥ 75% hair regrowth: 5/15 (33%)	AEs: asthenia, gingival hyperplasia/residual diastema, hypotrichosis, severe hypertension, atopy AE-related discontinuations: n = 1
Gupta et al. [38]	Non-randomized trial	Severe AA, AU, AT: > 5 patches on scalp	N = 6 AU: n = 3 AT: n = 1 AA: n = 2	CsA (6 mg/kg/d)	Mean 8 Yr (range 0.5–17)	Scalp hair regrowth > 90%	EOT (12 Wk) and after 3 Mth	Hair regrowth > 90%: EOT: 3/6 (50%) EOT + 3 Mth: 0/6 (0%); scalp cosmetically unacceptable in all patients	AEs: headache, dysesthesia, fatigue, diarrhea, erythematous rash, transient elevations in blood urea nitrogen, gingival hyperplasia, flushing, myalgias
Jang et al. [39]	Retrospective	Mild AA: < 50% scalp hair loss Severe AA: ≥ 50% scalp hair loss, including AT, AU	N = 88 Mild: n = 21 Severe: n = 67	CsA (50–400 mg/d) N = 51 BM (2–6 mg/day, 2 consecutive days per week) N = 37	NR	Response: > 50% hair regrowth	NR	Response Overall: CsA: 28/51 (55%) BM 14/37 (38%) Relapse after EOT: CsA: 2/51 (4%) BM: 1 (3%)	AE rates CsA: 29/51 (57%) BM: 27/37 (73%) AEs occurring in ≥ 5% pt: gastrointestinal symptoms, hypertrichosis, hypertension, weight gain, headache/dizziness, facial edema, acne, skin atrophy Nephrotoxicity reported in 3 pt receiving CsA
Shapiro et al. [40]	Non-randomized	Chronic severe AA: ≥ 95% scalp hair loss (AU, preuniversalis, AT)	N = 8	CsA (5 mg/kg/d) and prednisone (5 mg/d)	Mean 7.5 Yr	> 75% hair regrowth	24 Wk	> 75% hair regrowth: 2/8 (25%) Relapse after EOT 2/2 (100%)	‘Significant’ AEs in 4 patients included generalized edema, hypertension, abnormal liver function, abnormal lipid levels, hypertrichosis AE-related discontinuations: n = 3

All drugs administered orally unless otherwise mentioned

AA alopecia areata, AE adverse event, AT alopecia totalis, AU alopecia universalis, BID twice daily, BM betamethasone, CsA cyclosporine A, d days(s), EB eyebrow, EL eyelash, EOT end of therapy, Mth month(s), N total number of patients, n = number of patients in the category, NR not reported, PBO placebo, pt patient(s), RCT randomized controlled trial, SALT severity of alopecia tool, SALT₃₀ > 30% change in SALT score from BL, SAE serious AE, SLR systematic literature review, Yr year(s)

^†Including primary hair regrowth endpoint (where specified); other hair regrowth endpoints focus on SALT₅₀ and eyebrow and eyelash regrowth and relapse, where reported

^‡Definition of severity class not available