Background Clofarabine is a recently marketed drug used in paediatric patients with refractory acute lymphoblastic leukaemia (ALL) after at least two prior chemotherapy regimens. Its safety and effectiveness have not been evaluated in adults.
Purpose To evaluate the effectiveness and safety of clofarabine in adult and paediatric patients in a tertiary hospital and compare the results between the two populations.
Materials and methods Retrospective and descriptive study.
Inclusion criteria: all patients treated with clofarabine in the hospital. Data collected from clinical history and pharmacy database: patient age, diagnosis, prior relapses and number of cycles of clofarabine received.
Efficacy variables: disease remission (complete/partial) and the need for transplantation. Safety variables: all serious adverse reactions observed.
Results 16 patients were included: 10 adults and 6 children. Mean age was 33.4 (18–63) and 8 (3.7–13.5) years old, respectively.
Diagnoses were ALL (10 patients), acute myelogenous leukaemia (5) and lymphoblastic lymphoma (1). 2 (12.5%) patients received 2 cycles and the rest (87.5%) 1 cycle, all after the second relapse.
A complete response was attained in 33.3% of adults and 50% of children. 10% of adults had a partial response and 40% of adults and 33.3% children didn’t respond. 56.25% of patients needed subsequent transplantation.
The most significant adverse reactions in children were post-chemotherapy pancytopenia with infections (66.67%) and fulminant hepatic failure (16.67%). In adults pancytopenia (100%), bacteraemia, sepsis and severe infections (70%), hepatotoxicity (40%), central venous thrombosis (20%), severe gastrointestinal toxicity (20%), grade 4 mucositis (10%) and tumour lysis syndrome (10%) were observed.
13 patients died at the end point. 2 adults and 1 child died immediately after clofarabine treatment (due to post-chemotherapy hypovolaemic shock, tumour lysis syndrome and fulminant hepatic failure, respectively).
Conclusions Clofarabine effectiveness is low and bears a high risk of severe adverse effects in adults (especially infectious diseases or liver toxicity).
In paediatric patients, clofarabine is more effective and better tolerated.
No conflict of interest.
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