Background Clofarabine is a purine nucleoside antimetabolite, a second-generation antineoplastic indicated for the treatment of acute lymphoblastic leukaemia in paediatric patients (≤ 21 years) who have relapsed or are refractory after receiving at least two prior regimens and who have no other treatment options that provide a durable response.

Despite progress in leukaemia treatment, most children who relapse have a dismal prognosis. New approaches are needed.

Purpose To assess the effectiveness and safety of clofarabine in paediatric patients with refractory or relapsed acute leukaemia.

Materials and Methods This was an observational retrospective study. We included all paediatric patients diagnosed with acute leukaemia who received clofarabine as antineoplastic treatment during 2007–2011.

We used the computer programme Oncofarm for prescribing, preparation and validation of chemotherapy treatments and collected data for number of patients, age, sex, weight, height and treatment schemes lines administered prior to clofarabine.

In addition, we used medical records as a source of safety data regarding potential adverse reactions due to clofarabine.

Results During the study period clofarabine was administered to a total of six paediatric patients (4 boys and 2 girls) with a median age of 11.5 years (5–16 years).

They had received a median of 2 prior lines of treatment. Later treatment schedules used in 83.3% of these patients included 40 mg/m² clofarabine in combination with other chemotherapy drugs with a median of 2 administered cycles. In the rest (1/6) clofarabine was used at 52 mg/m² as monotherapy.

66.6% of patients achieved complete remission and 50% were transplanted.

Mucositis grade IV and pancytopenia were detected in two patients and palmar erythema in one patient. All patients had a transient transaminase increase.

Conclusions The administration of clofarabine allowed the team to reach transplantation in 50% of patients with acceptable toxicity, making it possible to expect durable responses.

No conflict of interest.