Effectiveness and safety of ciclosporin as therapy for autoimmune diseases of the liver in children and adolescents

Abstract

Summary Background : Conventional treatment for autoimmune hepatitis results in a significant percentage of failures and several, poorly tolerated, side‐effects. Therapy for autoimmune cholangitis and giant cell hepatitis associated with autoimmune haemolysis is poorly documented. Ciclosporin is a promising treatment for all of these diseases. Methods : We reviewed the records of 12 patients treated in our unit between 1987 and 2001. Eight had autoimmune hepatitis, two had autoimmune cholangitis and two had giant cell hepatitis. Indications for ciclosporin were treatment failure (four patients) and contraindications to/refusal of steroids (eight patients). Ciclosporin was administered in five untreated cases and in seven patients during relapse. The mean duration of ciclosporin administration was 35.6 months (8–89 months). The median follow‐up was 6.5 years (1.5–15 years). Results : All patients achieved complete remission in a median period of 4.5 weeks (2–12 weeks). No treatment withdrawal due to side‐effects occurred. Three patients required a combination of ciclosporin with conventional treatment due to severe liver function impairment. Tolerance to ciclosporin was excellent. A 20% transient elevation of serum creatinine occurred in one case, gingival hypertrophy in two and moderate hypertrichosis in two. Conclusions : Ciclosporin may be considered as a safe treatment for all autoimmune liver diseases and as an effective alternative for front‐line therapy.

Autoimmune hepatitis

“…CSA was generally well tolerated, and no renal insufficiency occurred. In another retrospective review 12 patients were treated with CSA: 8 patients with AIH, 2 with autoimmune cholangitis and 2 with giant cell hepatitis [186]. CSA was used in 4 with treatment failure and in 8 who refused steroids or had steroid contraindications.…”

Cyclosporine rescue therapy in autoimmune liver cirrhosis: A case report

“…Cyclosporine seemed to be a promising answer. Cyclosporine has been used in pediatric (7)(8)(9)(10)(11) and adult (12)(13)(14) autoimmune hepatitis patients with reported success and good tolerability. Malekzadeh et al (12) reported on a series of adult autoimmune hepatitis patients treated with cyclosporine.…”

“…Recent reports have suggested that cyclosporine may be effective in autoimmune hepatitis patients (7)(8)(9)(10)(11)(12)(13)(14). We present a case of successful cyclosporine use in a patient with autoimmune cirrhosis who was intolerant of azathioprine.…”

The management of childhood liver diseases in adulthood

“…Patients who fail azathioprine therapy or with refractory relapsing disease should be considered for treatment with mycophenolate mofetil, which can lead to remission in approximately 10% of children [101]. Calcineurin inhibitors (cyclosporine and tacrolimus) have also been used with good efficacy, but the data is limited to small numbers and limited long-term follow-up [102][103][104][105]. -Disease relapse.…”