Safety, efficacy and pharmacokinetics of palivizumab in off- label neonates, infants, and young children at risk for serious respiratory syncytial virus infection: a multicenter phase II clinical trial

被引:4
|
作者
Mori, Masaaki [1 ,2 ,16 ]
Yoshizaki, Kanako [3 ]
Watabe, Shinichi [3 ]
Ishige, Mika [4 ]
Hinoki, Akinari [5 ]
Kondo, Takuya [6 ]
Taguchi, Tomoaki [6 ,17 ]
Hasegawa, Hisaya [7 ]
Hatata, Tomoko [8 ]
Tanuma, Naoyuki [9 ]
Kirino, Kosuke [10 ]
Hirakawa, Akihiro [11 ]
Naruto, Takuya [12 ]
Imai, Minoru [13 ]
Koike, Ryuji [13 ,14 ]
Hosoi, Kenichiro [15 ]
Kusuda, Satoshi [15 ]
机构
[1] Tokyo Med & Dent Univ, Med Hosp, Dept Pediat, 1-5-45 Yushima,Bunkyo Ku, Tokyo 1138519, Japan
[2] St Marianna Univ, Sch Med, Dept Internal Med, Div Rheumatol & Allergol, 2-16-1 Sugao,Miyamae Ku, Kawasaki 2168511, Japan
[3] Kurashiki Cent Hosp, Dept Pediat, 1-1-1 Miwa, Kurashiki, Okayama 7108602, Japan
[4] Nihon Univ, Sch Med, Dept Pediat & Child Hlth, 1-6 Kanda Surugadai,Chiyoda Ku, Tokyo 1018309, Japan
[5] Nagoya Univ, Grad Sch Med, Dept Rare Intractable Canc Anal Res, 65 Tsurumai Cho,Showa Ku, Nagoya 4668550, Japan
[6] Kyushu Univ Hosp, Dept Pediat Surg, 3-1-1 Maidashi,Higashi Ku, Fukuoka 8128582, Japan
[7] Tokyo Womens Med Univ, Adachi Med Ctr, Div Neonatal Intens Care, 4-33-1 Kohoku,Adachi Ku, Tokyo 1168567, Japan
[8] Nagano Childrens Hosp, Dept Pediat Surg, 3100 Toyoshina, Azumino, Nagano 3998288, Japan
[9] Tokyo Metropolitan Fuchu Med Ctr Disabled, Dept Pediat, 2-9-2 Musashidai, Fuchu, Tokyo 1830042, Japan
[10] Natl Ctr Child Hlth & Dev, Clin Res Ctr, Dept Data Sci, 2-10-1 Ohkura,Setagaya Ku, Tokyo, Japan
[11] Tokyo Med & Dent Univ, Grad Sch Med & Dent Sci, Dept Clin Biostat, 1-5-45 Yushima,Bunkyo Ku, Tokyo 1138510, Japan
[12] Kanagawa Childrens Med Ctr, Clin Res Inst, 2-138-4 Mutsukawa,Minami Ku, Yokohama, Kanagawa 2328555, Japan
[13] Tokyo Med & Dent Univ, Hlth Sci Res & Dev Ctr, 1-5-45 Yushima,Bunkyo Ku, Tokyo 1138519, Japan
[14] Tokyo Med & Dent Univ Hosp, Clin Res Ctr, 1-5-45 Yushima,Bunkyo Ku, Tokyo 1138519, Japan
[15] Kyorin Univ, Fac Med, Dept Neurol, 6-20-2 Shinkawa, Mitaka, Tokyo 1818611, Japan
[16] Tokyo Med & Dent Univ Hosp, Dept Radiol, 1-5-45 Yushima,Bunkyo Ku, Tokyo 1138519, Japan
[17] Fukuoka Coll Hlth Sci, 2-15-1 Tamura,Sawara Ku, Fukuoka 8140193, Japan
来源
LANCET REGIONAL HEALTH-WESTERN PACIFIC | 2023年 / 39卷
关键词
Airway stenosis; Congenital esophageal atresia; Efficacy; Inherited metabolic disease; Neuromuscular disease; Palivizumab; Pediatric patient; Pulmonary hypoplasia; Respiratory syncytial virus infection; MONOCLONAL-ANTIBODY; MEDICAL DICTIONARY;
D O I
10.1016/j.lanwpc.2023.100847
中图分类号
R19 [保健组织与事业(卫生事业管理)];
学科分类号
摘要
Background Pediatric patients with certain rare diseases are at increased risk of severe respiratory syncytial virus (RSV) infection. However, the prophylactic use of anti-RSV antibody (palivizumab) in these patients is not indicated at present in Japan.Methods This first-in-the-world multicenter, uncontrolled, open-label, phase II clinical trial was carried out between 28 July 2019 and 24 September 2021 at seven medical institutions in Japan to investigate the efficacy, safety, and pharmacokinetics of palivizumab in 23 subjects recruited from among neonates, infants, or children aged 24 months or younger who had any of the following conditions: pulmonary hypoplasia, airway stenosis, congenital esophageal atresia, inherited metabolic disease, or neuromuscular disease. At least four continuous doses of palivizumab were administered intramuscularly at 15 mg/kg at intervals of 30 days.Findings Twenty-three enrolled subjects completed the study. No subject required hospitalization for RSV. Adverse events (AE) did not notably differ from the event terms described in the latest interview form. Five severe AEs required unplanned hospitalization, but resolved without RSV infection. Therapeutically effective concentrations of palivizumab were maintained throughout the study period. Interpretation Palivizumab might be well tolerated and effective in preventing serious respiratory symptoms and hospitalization due to severe RSV infection, indicating the prophylactic use in the pediatric patients included in this study.
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页数:12
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