Prevention of relapse after transplantation in children with high-risk leukemia by donor immune cell infusion: an open label, non randomized control study

Start Date01 Jun 2020

Sponsor / Collaborator

Beijing Jingdu Children's Hospital Co., Ltd.

ChiCTR1900021288

/ SuspendedNot Applicable

Study for mTOR Inhibitor as Adjunctive Therapy in children With Cerebral Adrenoleukodystrophy

Start Date11 Feb 2019

Sponsor / Collaborator

Beijing Jingdu Children's Hospital Co., Ltd.

100 Clinical Results associated with Beijing Jingdu Children's Hospital Co., Ltd.

0 Patents (Medical) associated with Beijing Jingdu Children's Hospital Co., Ltd.

Literatures (Medical) associated with Beijing Jingdu Children's Hospital Co., Ltd.

02 Mar 2025·Zhonghua er ke za zhi = Chinese journal of pediatrics

[Analysis of 21 cases of Barth syndrome in children].

Article

Author: Liu, Z W ; Han, L ; Lyu, Z Y ; Xiao, Y Y ; Yu, W ; Wang, Z Y ; Ding, W W

Objective: To investigate the clinical manifestations, treatment, and outcomes of Barth syndrome (BTHS). Methods: A retrospective analysis was conducted on 21 pediatric patients diagnosed with BTHS between January 2010 and December 2023 at Beijing Children's Hospital, Beijing Anzhen Hospital, and Beijing JingDu Children's Hospital. Clinical data including gender, age at onset, initial symptoms, clinical manifestations, personal history, family genetic history, and laboratory tests (neutrophil count, echocardiography, electrocardiogram and genetic testing) were reviewed. Results: All the 21 patients were male, with the age of onset at 4.1 (1.1, 9.3) months. Main clinical manifestations included heart failure (18 cases), neutropenia (16 cases), respiratory symptoms (15 cases), 3-methylpentenediuria (7 cases),develop retardation (8 cases), gastrointestinal symptoms (7 cases), fatigue and anorexia (6 cases), and recurrent infection (2 cases). Electrocardiogram abnormalities included ST changes (18 cases), flattened T wave and low voltage of limb leads (2 cases), and abnormal Q waves in lead Ⅰ and avL (1 case). Echocardiographic features showed increased trabeculation, interventricular septum and left ventricular wall thickening, and left ventricular enlargement with reduced ejection fraction. Genetic testing identified TAZ gene variations in all 21 patients: 11 missense mutations, 2 nonsense mutations, 2 frameshift mutations, 2 whole code mutations, 2 exon deletions, 1 splicing mutation, and 1 synonymous mutation. Fifteen mutations were maternally inherited, 2 were de novo, and 4 lacked verified variant origin.In terms of treatment, all 18 patients with heart failure received routine heart failure treatment, of whom 11 patients also received intravenous immunoglobulin and corticosteroids. After the follow-up of 91.0 (75.5, 109.5) months, 15 of the 18 patients showed restoration of cardiac function after 4.5 (3.0, 9.8) months of treatment, with one case of significant improvement, while 2 cases suddenly died. Conclusions: BTHS predominantly affects males with early onset, mainly characterized by abnormal cardiac structure and function, along with clinical features including fatigue, delayed growth and development, and neutropenia. Early diagnosis and intervention, including heart failure treatment, intravenous immunoglobulin, and corticosteroids, can lead to significant improvement in cardiac function, though sudden death remains a risk.

01 Feb 2025·Annals of Hematology

Belumosudil in pediatric patients with chronic graft-versus-host disease after failed multi-line therapy: a case series

Article

Author: Liu, Zhouyang ; You, Jianhua ; Fu, Bin ; Hu, Jiong ; Chen, Wenting ; Wang, Zhi ; Xing, Tingting

Belumosudil is a selective small molecule inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2) indicated for patients with glucocorticoid-refractory chronic graft-versus-host disease (cGVHD). Despite its approval for ages 12-18, there is limited pediatric data available. This case series presents three 12-year-old patients with severe cGVHD who had failed multiple lines of therapy. Case #1 received treatment for 210 days with belumosudil, prednisone, cyclosporine A, mycophenolate mofetil, and ruxolitinib. Initial assessments showed skin and joint fascia involvement (National Institutes of Health score 3), along with oral cavity, ocular, and pulmonary involvement (score 2). Following treatment, all affected organs demonstrated at least a partial response (PR), with an overall assessment of PR. Case #2 was treated for 205 days with belumosudil, tacrolimus, and ruxolitinib. Baseline assessments indicated involvement of the skin and joint fascia (score 3), and the oral cavity and eyes (score 2). Most organs achieved PR or complete response (CR), resulting in an overall PR. Case #3 underwent 121 days of therapy with belumosudil, prednisone, and tacrolimus, showing similar baseline organ involvement as Case #2. The treatment resulted in an overall PR, with improvement noted in the skin, oral cavity, eyes, and joint fascia. The Lee cGVHD symptom scale scores improved meaningfully for all patients over time. There was no recurrence of the primary disease or any fatalities. Adverse events were limited to grade 1-2 severity. This case series indicates that belumosudil may be effective in 12-year-old pediatric patients with severe, multi-organ cGVHD refractory to multiple treatments. The findings suggest that belumosudil-based regimens can be feasible and well-tolerated in this population, providing preliminary evidence for its potential therapeutic effects in clinical management.

01 Apr 2024·Maturitas

Practice guideline on ovarian tissue cryopreservation and transplantation in the prevention and treatment of iatrogenic premature ovarian insufficiency

Review

Author: Gu, Muqing ; Wang, Liqun ; Wang, Jinping ; Wang, Yue ; Hu, Zhuoying ; Mueck, Alfred O ; Ma, Fei ; Sun, Yuan ; Lin, Yuan ; Ni, Rong ; Jin, Jing ; Wang, Fuling ; Tian, Ying ; Yang, Xiaomin ; Shi, Dongmei ; Xu, Che ; Ju, Rui ; Huang, Hefeng ; Xu, Liangzhi ; Wu, Yurui ; Jin, Quanfang ; Li, Lianfang ; Tian, Qinjie ; Cheng, Yanxiang ; Xu, Binghe ; Yang, Caihong ; Jin, Fengyu ; Qin, Maoquan ; Ye, Xiyang ; Cheng, Jiaojiao ; Li, Yan ; Kong, Weimin ; Zhu, Tianyuan ; Zhang, Wei ; Yin, Chenghong ; Cheng, Linghui ; Wei, Wei ; Zou, Lin ; Hao, Min ; Huang, Yanhong ; Cao, Yunxia ; Ruan, Xiangyan ; Huang, Xiaojun ; Li, Peiling ; Min, Aiping ; Luo, Guangxia ; Zhang, Siyou ; Wu, Ruifang ; Wang, Gang ; Liu, Rong ; Du, Juan ; Li, Xiaodong ; Dai, Yinmei ; Niu, Jumin ; Ren, Fangying ; Li, Yanglu

Premature ovarian insufficiency (POI) refers to the decline of ovarian function before the age of 40. POI causes a reduction in or loss of female fertility, accompanied by different degrees of menopausal symptoms, which increases the risk of chronic diseases related to early menopause and seriously affects patients' quality of life and health. It is conservatively estimated that at least one million prepubertal girls and women of reproductive age in China are at risk of iatrogenic POI caused by radiotherapy and chemotherapy every year. With the development of medical technology and the breakthrough of scientific and technological advances, preventing and treating iatrogenic POI have become possible. International and national guidelines consider cryopreserved ovarian tissue transplantation to be the most promising method of preserving the ovarian function and fertility of prepubertal girls and women of reproductive age who cannot delay radiotherapy and chemotherapy. In order to guide the clinical application of ovarian tissue cryopreservation and transplantation technology in China, the Guideline Working Group finally included 14 scientific questions and 18 recommendations through a questionnaire survey, field investigation, and consultation of a large number of Chinese and English literature databases in order to provide a reference for colleagues in clinical practice.

100 Deals associated with Beijing Jingdu Children's Hospital Co., Ltd.

100 Translational Medicine associated with Beijing Jingdu Children's Hospital Co., Ltd.

Corporation Tree

Boost your research with our corporation tree data.

view full example data

Pipeline

Pipeline Snapshot as of 20 Dec 2025

No data posted

Deal

Boost your decision using our deal data.

view full example data

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Profit

Explore the financial positions of over 360K organizations with Synapse.

view full example data

Grant & Funding(NIH)

Access more than 2 million grant and funding information to elevate your research journey.

view full example data

Investment

Gain insights on the latest company investments from start-ups to established corporations.

view full example data

Financing

Unearth financing trends to validate and advance investment opportunities.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Beijing Jingdu Children's Hospital Co., Ltd.

Overview

Related

Corporation Tree

Pipeline

Pipeline Snapshot as of 20 Dec 2025

Deal

Translational Medicine

Profit

Grant & Funding(NIH)

Investment

Financing