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Annual Report 2024

Department of Pediatric Oncology

Toru Mukohara, Ako Hosono

Introduction

 Since its establishment in December 2011, the Department of Pediatric Oncology has been dedicated to the comprehensive management and medical treatment of solid tumors commonly diagnosed in children and adolescents, known as pediatric and AYA (adolescent and young adult) cancers. We collaborate closely with related departments, including medical oncology, orthopedic oncology, urology, radiation oncology, the AYA support team, and the neuro-oncology and ocular oncology units at the central hospital, to provide multidisciplinary treatment.

 Through active collaboration with the AYA support team, we have also enhanced the psychosocial and lifestyle support available to our patients, allowing for more comprehensive, patient-centered care.

 A distinctive feature of our department is our strong partnership with medical oncology, which enables us to provide seamless care for a wide age range-from older children in late elementary school to adolescents and young adults. In addition to treatment, we emphasize holistic care for pediatric and AYA patients, including educational support, family support (for parents, siblings, and children of patients), and long-term follow-up for late complications.

 Since April 2016, pediatric cancer patients have been eligible for insurance-covered proton therapy in Japan. This has resulted in a steady increase in patients from both Japan and abroad, particularly from China, seeking advanced proton beam therapy at our hospital. In collaboration with the Department of Radiation Oncology, we have established a specialized and safe treatment environment for pediatric patients. In recent years, further improvements in collaboration and technology have enabled us to deliver more comprehensive and sophisticated treatment strategies. Our main areas of focus include brain tumors and sarcomas such as rhabdomyosarcoma and Ewing sarcoma, which develop in the head, neck, and torso. For sarcomas, radiation therapy is commonly combined with chemotherapy, facilitated by our outpatient treatment center, allowing for both efficacy and quality of life.

The Team and What We Do

 The pediatric outpatient department treats newly diagnosed patients and patients undergoing outpatient chemotherapy every Wednesday and provides follow-up treatment for patients who have completed an intensive course of treatment. Flexible arrangements are made for outpatient treatment of patients on days other than Wednesdays. Cancer patients of the AYA generation are treated jointly with the Department of Oncology every Monday, Wednesday and Friday. Ward rounds and conferences are held each morning jointly with the Medical Oncology group. Sarcoma conferences are held every other Tuesday. For patients undergoing proton beam therapy, a conference is always held with the participation of the radiation therapy department, nurses, and technicians.

Research Activities

 In addition to providing standard treatment for newly diagnosed patients, we have also established a system that allows us to provide clinical trial treatment for patients with relapsed or refractory disease, including the use of drugs outside of the approved indications, and we accept patients from other medical institutions.

Future Prospects

 There are three major missions in the Department of Pediatric Oncology of the National Cancer Center Hospital East (NCCHE), as follows:

1) To provide state-of-the-art treatment for AYA patients in collaboration with the Medical Oncology group and AYA support team

2) To develop new treatments for pediatric cancer by sharing agents and knowledge with the Clinical Development Center

3) To provide less toxic proton-beam radiation therapy as one of the three proton centers for children in Japan

 All three activities are in progress and several projects have already started.

List of papers published in 2024

Journal

1. Tsuchiya K, Akisue T, Ehara S, Kawai A, Kawano H, Hiraga H, Hosono A, Hutani H, Morii T, Morioka H, Nishida Y, Oda Y, Ogose A, Shimose S, Yamaguchi T, Yamamoto T, Yoshida M. Japanese orthopaedic association (JOA) clinical practice guideline on the management of primary malignant bone tumors - Secondary publication. Journal of orthopaedic science, 30:1-17, 2025

2. Iwata H, Naito Y, Hattori M, Yoshimura A, Yonemori K, Aizawa M, Mori Y, Yoshimitsu J, Umeyama Y, Mukohara T. Safety and pharmacokinetics of vepdegestrant in Japanese patients with ER+ advanced breast cancer: a phase 1 study. International journal of clinical oncology, 30:72-82, 2025

3. Takahashi K, Uozumi R, Mukohara T, Hayashida T, Iwabe M, Iihara H, Kusuhara-Mamishin K, Kitagawa Y, Tsuchiya M, Kitahora M, Nagayama A, Kosaka S, Asano-Niwa Y, Seki T, Ohnuki K, Suzuki A, Ono F, Futamura M, Kawazoe H, Nakamura T. Proton Pump Inhibitors and Cyclin-Dependent Kinase 4/6 Inhibitors in Patients With Breast Cancer. The oncologist, 29:e741-e749, 2024

4. Bardia A, Krop IE, Kogawa T, Juric D, Tolcher AW, Hamilton EP, Mukohara T, Lisberg A, Shimizu T, Spira AI, Tsurutani J, Damodaran S, Papadopoulos KP, Greenberg J, Kobayashi F, Zebger-Gong H, Wong R, Kawasaki Y, Nakamura T, Meric-Bernstam F. Datopotamab Deruxtecan in Advanced or Metastatic HR+/HER2- and Triple-Negative Breast Cancer: Results From the Phase I TROPION-PanTumor01 Study. Journal of clinical oncology, 42:2281-2294, 2024

5. Mukohara T, Park YH, Sommerhalder D, Yonemori K, Hamilton E, Kim SB, Kim JH, Iwata H, Yamashita T, Layman RM, Mita M, Clay T, Chae YS, Oakman C, Yan F, Kim GM, Im SA, Lindeman GJ, Rugo HS, Liyanage M, Saul M, Le Corre C, Skoura A, Liu L, Li M, LoRusso PM. Inhibition of lysine acetyltransferase KAT6 in ER(+)HER2(-) metastatic breast cancer: a phase 1 trial. Nature medicine, 30:2242-2250, 2024

6. Mukohara T, Park YH, Sommerhalder D, Yonemori K, Hamilton E, Kim SB, Kim JH, Iwata H, Yamashita T, Layman RM, Mita M, Clay T, Chae YS, Oakman C, Yan F, Kim GM, Im SA, Lindeman GJ, Rugo HS, Liyanage M, Saul M, Le Corre C, Skoura A, Liu L, Li M, LoRusso PM. Author Correction: Inhibition of lysine acetyltransferase KAT6 in ER(+)HER2(-) metastatic breast cancer: a phase 1 trial. Nature medicine, 30:2371, 2024

7. Fukuda M, Mukohara T, Kuwata T, Sunami K, Naito Y. Efficacy of Trametinib in Neurofibromatosis Type 1-Associated Gastrointestinal Stromal Tumors: A Case Report. JCO precision oncology, 8:e2300649, 2024

8. Nakao T, Harano K, Wakabayashi M, Naito Y, Tanabe H, Mukohara T. Efficacy of a platinum-based chemotherapy rechallenge for platinum-sensitive recurrence after PARP inhibitor maintenance. Gynecologic oncology reports, 55:101482, 2024

Book

1. Nakajima H, Mukohara T. Understanding mechanisms of resistance to HER2-targeted therapies in HER2-positive breast cancer. In: Bonavida B (ed), Overcoming Cancers Resistant to HER-2 Antibodies: Breaking Tolerance to Antibody-Mediated Immunotherapy, United Kingdom, Academic Press, pp 45-56, 2024