Real-Life Management of Children and Adolescents with Chronic Myeloid Leukemia: The Italian Experience

Abstract

Background: To date, no data on the adherence to specific guidelines for children with chronic myeloid leukemia (CML) in chronic phase (CP) have been reported. Methods: Since 2001, guidelines for treatment with imatinib mesylate (IM) and monitoring in patients younger than 18 years with CP-CML have been shared with 9 pediatric referral centers (P centers) and 4 reference centers for adults and children/adolescents (AP centers) in Italy. In this study, the adherence to these guidelines was analyzed. Results: Thirty-four patients with a median age of 11.4 years and 23 patients with a median age of 11.0 years were managed at 9 P and at 4 AP centers, respectively. Evaluations of bone marrow (BM) and/or peripheral blood (PB) were available for more than 90% of evaluable patients. Cytogenetics and molecular monitoring of PB were more consistently performed in AP centers, whereas molecular analysis of BM was carried out more frequently in P centers. Before 2009, some patients who responded to IM underwent a transplantation, contrary to the guidelines’ recommendations. Conclusions: Our experience shows that having specific guidelines is an important tool for an optimal management of childhood CP-CML, together with exchange of knowledge and proactive discussions within the network.

Features and outcome of chronic myeloid leukemia at very young age: Data from the International Pediatric Chronic Myeloid Leukemia Registry

“…In all eligible patients (missing data from two patients), the CHR rate achieved by IMA treatment was 65% (n = 13/20). This rate is lower compared to previous reports with older children who achieved CHR in more than 95% of cases 22,23 . The proportion of children initially presenting in chronic phase and achieving CHR according to the ELN citeria at 3 months of therapy was 59% (n = 10/17).…”

“…reported 46% and 61% CCyR rate at 6 and 12 months, respectively, in newly diagnosed CML patients treated with IMA 260 mg/sqm/day 23 . The Italian experience administering a daily IMA dosage of 340 mg/m 2 resulted in a 29.6 % CCyR rate at 3 months 22 . However, none of the children in the current study achieved CCyR earlier than 4 months after initiation of IMA treatment.…”

“…This rate is lower compared to previous reports with older children who achieved CHR in more than 95% of cases. 22,23 The proportion of children initially presenting in chronic phase and achieving CHR according to the ELN citeria at 3 months of therapy was 59% (n = 10/17). Also the three cases diagnosed in advanced phase all achieved CHR at 3 months.…”

Efficacy and Safety of Imatinib in Paediatric CML – A Single Centre Study

“…An improved conditioning regimen of allogeneic-HSCT with reduced-intensity stem cell transplantation could minimize the post-transplant mortality (19). Since CML remains a rare, but also a debilitating disease in the paediatric population, guidelines for treating paediatric CML need to be continuously optimized and incorporate the therapeutic experience stemming from adult CML (27). Clinical experience from the adult population may guide the therapeutic decision making process, however, care should be taken in terms of drug formulation, pharmacokinetics and compliance when applying the treatment to the fragile paediatric age group (28).…”

Chronic Myeloid Leukemia in Children: Immune Function and Vaccinations

“…The authors concluded that imatinib therapy in CML is associated with a low incidence of VZV infection, does not disseminate, responds well to therapy, and does not mandate a recommendation for herpes zoster prophylaxis in patients with CML [ 32 ]. In pediatric trials of imatinib, no unusual cases of VZV infection were reported [ 8 , 9 , 33 ]. This is in contrast to the experience with other pediatric cancers or other diseases requiring immunosuppressive therapies, which have the risk for a potentially fatal course of VZV infection.…”