hrp0097p1-289 | GH and IGFs | ESPE2023

Long-term effectiveness and safety of growth hormone therapy in Japanese children with short stature due to Noonan syndrome (NS): real-world data

Muroya Koji , Kawai Masanobu , Yamagishi Hiroyuki , Endo Takaaki , Pietropoli Alberto , Horikawa Reiko

Norditropin® was approved for children with short stature due to NS in Japan in 2017. The aim of this post-marketing surveillance study was to evaluate long-term safety and effectiveness of Norditropin® for the approved indication. This real-world non-interventional study (NCT03435627) was conducted February 2018–January 2022. Seventy patients enrolled: 35 received Norditropin® after study initiation (new patients); 35 were previously ra...

hrp0092p1-243 | Multisystem Endocrine Disorders | ESPE2019

A Nation-Wide Questionnaire Survey Targeting Japanese Pediatric Endocrinologists Regarding Transitional Care in Pediatric and Adolescent Cancer Patients

Miyoshi Yoko , Yorifuji Tohru , Yokoya Susumu , Nagasaki Keisuke , Kawai Masanobu , Ishiguro Hiroyuki , Okada Satoshi , Kanno Junko , Takubo Noriyuki , Muroya Koji , Ito Junko , Horikawa Reiko , Shimizu Chikako , Ozono Keiichi

Background: While existing guidelines recommend long-term follow-up of childhood cancer survivors (CCS), transitional care among pediatric and adult endocrinologists has not been established in Japan.Objective and Hypotheses: To know the present situation and to cultivate a better understanding, we had conducted a nation-wide survey targeting Japanese pediatric endocrinologists.Method</stro...

hrp0092p3-54 | Diabetes and Insulin | ESPE2019

Relationship Between Chloride Infusion and Base Excess in Initial Treatment of Pediatric Diabetic Ketoacidosis

Sawano Kentaro , Nagamatsu Fusa , Shimura Kazuhiro , Abe Yuki , Izumita Yukie , Ogawa Yohei , Komatsu Nagisa , Takishima Shigeru , Nakamura Akie , Nyuzuki Hiromi , Yamaguchi Takeshi , Muroya Koji , Watanabe-Yamamoto Sayaka , Nagasaki Keisuke , Saitoh Akihiko , Hasegawa Yukihiro

Background: In initial treatment of DKA, volume expansion should begin with 0.9% saline to restore the peripheral circulation. The use of large amounts of chloride-rich/bicarbonate-free fluids may cause the rapid development of hyperchloremic metabolic acidosis, which is described in ISPAD Clinical Practice Consensus Guidelines 2018.The severity of DKA, defined by pH, HCO3- Base Excess (BE), is one of the factor...

hrp0095fc2.4 | Bone, Growth Plate and Mineral Metabolism | ESPE2022

Patient-reported outcomes from a randomized open-label phase 3 trial comparing burosumab vs conventional therapy in children with X-linked hypophosphatemia: results from the 24-week treatment extension period

Padidela Raja , Whyte Michael P , Glorieux Francis H , Munns Craig F , Ward Leanne M , Nilsson Ola , Portale Anthony A , Simmons Jill H , Namba Noriyuki , Cheong Hae Il , Pitukcheewanont Pisit , Sochett Etienne , Högler Wolfgang , Muroya Koji , Tanaka Hiroyuki , Gottesman Gary S , Biggin Andrew , Perwad Farzana , Williams Angela , Nixon Annabel , Sun Wei , Chen Angel , Skrinar Alison , Imel Erik A

In a randomized open-label phase 3 trial in 62 children (1–12 years) with X-linked hypophosphatemia (XLH) (NCT 02915705), switching from conventional therapy (oral phosphate plus active vitamin D) to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved serum phosphate concentration, rickets, lower-extremity deformities, growth, mobility, and patient-reported outcomes (PROs) at 64 weeks. Children in Europe, USA, Canada, and Australia wh...