Concurrent Multiple Sclerosis and Primary Sclerosing Cholangitis Treated with Oral Cladribine
Carlos Guevara-Oliva1, Eduardo Villa1, Antonio Piottante2, Gonzalo Carrasco Avino2, Rodrigo Naves1
1Universidad de Chile, 2Clínica Las Condes
Objective:
To present a patient diagnosed with concomitant Primary Sclerosing Cholangitis (PSC) and Multiple Sclerosis (MS) who underwent treatment with oral cladribine and experienced resolution of both liver and neurological symptoms, along with brain imaging stability and normalization of liver laboratory parameters during a 2-year follow-up period.
Background:
PSC is a chronic hepatic autoimmune disorder of unknown etiology thought to be mediated by biliary autoreactive lymphocytes. Liver transplantation is the only curative treatment. MS is an immune-mediated demyelinating disease affecting the central nervous system. Cladribine is an established oral chemotherapy approved for MS that, when activated intracellularly, preferentially depletes T and B lymphocytes, followed by immune reconstitution with improved immune tolerance. Co-occurrence of PSC and MS has only been reported once.
Results:
A 33-year-old female presented with an acute onset of blurred vision in her right eye and painful eye movement. The MRI showed hyperintense lesions in the periventricular and juxtacortical white matter and a lesion extending from C4 to C5. She was diagnosed with MS and started on glatiramer acetate. During the subsequent years, she developed jaundice, fatigue, pruritus, and recurrent elevated transaminase levels, with ultrasound findings consistent with steatohepatitis. Despite a comprehensive etiological study, no underlying cause was identified. Liver needle biopsy revealed concentric "onionskin" bile duct fibrosis, indicative of PSC. Ursodeoxycholic acid therapy did not result in any symptom improvement. In addition, she experienced two MS relapses. Oral cladribine (1.75 mg/kg/course PO) was given monthly in two cycles. Pruritus, fatigue, nausea, and vomiting subsided, and liver function tests normalized during two-year follow-up. Furthermore, she presented no relapses and no new brain lesions on the MRI.
Conclusions:
This patient presented concomitant PSC and MS treated with oral cladribine. By targeting lymphocyte-mediated pathogenesis, cladribine alleviated both the liver and neurological symptoms, offering a therapeutic approach for PSC and MS.
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