Retrospective study of children with chronic myeloid leukemia treated in Belgium between 2000 and 2021

Introduction: Chronic myeloid leukemia (CML) in children and adolescents is a rare disease with an annual incidence of one case per million. The condition is characterized by the translocation t(9;22)(q34;q11.2) resulting in a BCR-ABL1 fusion oncoprotein localized on the newly formed Philadelphia (Ph) chromosome. With the current retrospective study, we aimed to identify the clinical and biological characteristics of Belgian CML patients up to 18 years treated in Belgium between 2000 and 2021, as well as to evaluate the response to treatment, potential long-term side effects, and prognosis. Methodology: Children and adolescents up to 18 years of age treated for CML in eight Belgian pediatric hemato-oncology centers between 2000 and 2021 were included in the present study as part of an international registry study (I-CML-Ped Study). The data of the pediatric CML patients were collected in a Belgian CML registry on case report forms (CRFs) after which they were inserted into a database. IBM SPSS Statistics was used for statistical analysis of the data. Survival curves were made using the Kaplan-Meier method. Results: A total of 30 pediatric CML patients treated between 2000 and 2021 in Belgium were included for data analysis. The population consisted of 10 boys and 20 girls with a mean age of 9 years (range 1-16 years). The mean follow-up time was 99 months with a range of 8 to 247 months. The first symptoms of CML were from most to least common: asthenia, weight loss, abdominal pain, fever, and bleeding. The spleen was palpable in 22 patients with a mean of 10 cm below the costal margin (range 1-20 cm). Twenty-nine patients were in the chronic phase (CML-CP) at diagnosis, while one patient was in the blast phase (CML-BC). In the pre-TKI era (before 2004), the majority of the patients were treated with an HSCT preceded by Hydrea. After 2004, the standard first treatment was imatinib. The overall survival (OS) of all included patients was 96.7%. Conclusion: In general, the clinical and biological characteristics of the Belgian pediatric CML population are in line with the literature. Although the outcome is excellent, it is essential to consider the long-term side effects of TKI treatment and a hematopoietic stem cell transplantation (HSCT) and weigh the advantages and disadvantages of both treatments

Growth Hormone Replacement Therapy Seems to Be Safe in Children with Low-Grade Midline Glioma: A Series of 124 Cases with Review of the Literature

There is little scientific evidence regarding the safety of GHRT in LGG, where GH deficiency is common. Purpose: to compare the recurrence rate in children with midline LGG, depending on whether or not they have received GHRT, in order to assess its impact on the risk of tumor recurrence. Methods: This bicentric retrospective study included 124 patients under the age of 18 who were diagnosed with a midline low-grade glial tumor between 1998 and 2016. We also reviewed literature on this subject. The main outcome measure was tumor relapse, demonstrated by brain MRI. Results: There were 17 patients in the GH-supplemented group (14%) and 107 patients in the non-supplemented group (86%). Relapse occurred in 65 patients (45.5%); 7 patients died (4.9%); no deaths occurred in patients receiving GHRT. Two patients developed a second tumor (1.4%), none of which had received GHRT. Relapse concerned 36.4% of patients without GHRT and 52.9% of patients with GHRT. The difference was not statistically significant between the two groups (p = 0.3). Conclusion: GHRT does not lead to a statistically significant increase in risk of relapse for pediatric midline low-grade pediatric glioma in our cohort. Although these results appear reassuring, future natural history or prospective studies should be done to ascertain these findings. Nevertheless, these reassuring data regarding GHRT are in agreement with the data in the current literature

Growth Hormone Replacement Therapy Seems to Be Safe in Children with Low-Grade Midline Glioma: A Series of 124 Cases with Review of the Literature

There is little scientific evidence regarding the safety of GHRT in LGG, where GH deficiency is common. Purpose: to compare the recurrence rate in children with midline LGG, depending on whether or not they have received GHRT, in order to assess its impact on the risk of tumor recurrence. Methods: This bicentric retrospective study included 124 patients under the age of 18 who were diagnosed with a midline low-grade glial tumor between 1998 and 2016. We also reviewed literature on this subject. The main outcome measure was tumor relapse, demonstrated by brain MRI. Results: There were 17 patients in the GH-supplemented group (14%) and 107 patients in the non-supplemented group (86%). Relapse occurred in 65 patients (45.5%); 7 patients died (4.9%); no deaths occurred in patients receiving GHRT. Two patients developed a second tumor (1.4%), none of which had received GHRT. Relapse concerned 36.4% of patients without GHRT and 52.9% of patients with GHRT. The difference was not statistically significant between the two groups (p = 0.3). Conclusion: GHRT does not lead to a statistically significant increase in risk of relapse for pediatric midline low-grade pediatric glioma in our cohort. Although these results appear reassuring, future natural history or prospective studies should be done to ascertain these findings. Nevertheless, these reassuring data regarding GHRT are in agreement with the data in the current literature