Childhood Chronic Myeloid Leukemia in Kuwait

CHILDHOOD CHRONIC MYELOID LEUKEMIA IN THE SOLE NATIONAL SERVICE FOR CHILDHOOD CANCER IN THE STATE OF KUWAIT MAHA JASSIM BOURUSLY, MOHAMMAD ADIL, MONA BOURHAMA, NISREEN KHALIFA, HUBERT MOTTL, SAHAR KALEEFA SC-KWT-CP-00002 Aug 2020 Amgen Proprietary - For Internal Use Only

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NBK CHILDREN'S CANCER HOSPITAL, KUWAIT Amgen Confidential. For Internal Use Only. Class V: This material is for your information and/or training only. It cannot be used in connection with any sales activities, and may not be shown to members of the Healthcare Community. Amgen Proprietary - For Internal Use Only 4

INTRODUCTION Chronic myelogenous leukemia (CML) is a rare disease in children On an average NBK children specialty hospital in Kuwait (the sole children cancer center in Kuwait) receives yearly one or 2 children with CML. There is little evidence of biological differences between CML in children and adults in terms of disease presentation and progression, which should be considered when treating pediatric patients with CML. children. Amgen Confidential. For Internal Use Only. Class V: This material is for your information and/or training only. It cannot be used in connection with any sales activities, and may not be shown to members of the Healthcare Community. 5 Amgen Proprietary - For Internal Use Only

INTRODUCTION The goal of treatment in children should be cure rather than suppression of disease. However, the issue of how long the treatment should be continued still remains unsolved. Prognostic factors applicable in adult CML to identify high-risk patients at diagnosis are not established for children. Because of the low incidence of CML in children, only a few pediatric studies have evaluated efficacy of treating this disease with TKI therapy in children. Amgen Proprietary - For Internal Use Only Amgen Confidential. For Internal Use Only. 6

METHOD This is a retrospective study of all children who presented with CML. The inclusion criteria were all children with CML who presented during the last ten years. Amgen Proprietary - For Internal Use Only Amgen Confidential. For Internal Use Only. 7

RESULTS Eleven children presented to our center with CML in the last decade. At presentation the majority presented with: leukocytosis (8/11) splenomegaly. The median size of the spleen was 11 cm below the costal margin (range, 2-16 cm) The median baseline WBC was 290 109/L, and for platelets were 617 109/L Amgen Proprietary - For Internal Use Only Amgen Confidential. For Internal Use Only. 8

RESULTS Some children initially required leukapharesis (3/11 = 27%) and /or received hydroxyurea (8/11= 72%) Almost all responded well to first line TKI Imatinib at 340 mg/m2. Some children had to be shifted to second line TKI later, either for failing to reach targeted response or development of drug related complications. None required BMT, and two were discharged to adult care. Amgen Proprietary - For Internal Use Only Amgen Confidential. For Internal Use Only. 9

DISCUSSION Children with CML-CP tend to have clinical presentations with more aggressive features, although the impact on prognosis is debatable. In our study many children presented with what looked like an aggressive disease; presented with high leukocytosis, and or splenomegaly. These children received initially hydroxyurea and few required leukapharesis; though all responded well to TKI. Amgen Proprietary - For Internal Use Only Amgen Confidential. For Internal Use Only. 10

DISCUSSION The median size of the spleen, baseline white blood cell count and platelets were higher than adults in comparison to other studies, however this had no effect on response to outcome or survival. Some children had to be shifted to second line TKI, either for lack of response or development of drug related complications. None of the children developed acute blastosis AP, or had their medication stopped. Amgen Proprietary - For Internal Use Only Amgen Confidential. For Internal Use Only. 11

CONCLUSION CML is a rare disease in children. Effort should be directed to have clinical studies in our region of the world for children with CML. Amgen Proprietary - For Internal Use Only Amgen Confidential. For Internal Use Only. 12

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