Effect of allogeneic hematopoietic stem cell transplantation for chronic granulomatous disease in children: A multicentre, retrospective cohort study in China

Article

Author: Wang, Ping ; Lu, Wei ; Meng, Yan ; Qian, Xiaowen ; Chen, Jing ; Luo, Changying ; Tang, Xiangfeng ; Si, Yingjian ; Zhou, Chen ; Yu, Jie ; Zhai, Xiaowen ; Dou, Ying

Chronic granulomatous disease (CGD) in children is a rare primary immunodeficiency disorder that can lead to life-threatening infections and inflammatory complications. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is increasingly being used to treat severe CGD in children. We conducted a multicenter retrospective analysis of children with CGD who were treated with allo-HSCT at four pediatric hematopoietic stem cell transplant centers in China from September 2005 to December 2019. The study included a total of 171 patients (169 males and 2 females). The median age at the time of transplantation was 6.1 (0-16.4) years. Among them, 154 patients had X-linked recessive inheritance caused by CYBB gene mutations, 12 patients were autosomal recessive, 1 patient had DNAH11 and HYDIN gene mutations, and 4 patients had no gene mutations. The median follow-up period was 36.3 (1.9-79) months. All participating patients were applied to myeloablative conditioning (MAC) regimens. The rates of OS, EFS, and GEFS within three years were 87.5%, 85.3%, and 75.2%, respectively. The total graft failure and the total mortality rate were 5.3% and 11.1%. The cumulative incidence of acute GVHD was 53.8% and the incidence of chronic GVHD was 12.9%, The incidence of chronic GVHD was higher for patients who received unrelated donor cord blood stem cell transplantation (UD-CB) (P = 0.001). Chronic GVHD and coinfections are the risk factors for OS and EFS in patients with CGD after receiving allo-HSCT. UD-CB is a risk factor for EFS and the presence of pneumonia before transplantation is a risk factor for OS. In conclusion, through this study, we have demonstrated that allo-HSCT has excellent efficacy in the treatment of CGD in children, especially, RD-haplo is associated with a lower rate of graft failure incidence and mortality than the treatment modalities of other donor type. Therefore, allo-HSCT is strongly recommended when a well-matched donor is available. If a well-matched donor is not available, the HLA-mismatched donor should be carefully evaluated, and the conditioning regimen modified accordingly.

01 Jan 2024·Pesticide Biochemistry and Physiology

Molluscicidal activity of calcium borate nanoparticles with kodom ball-flower structure on hematological, histological and biochemical parameters of Eobania vermiculata snails

Article

Author: Elkady, Elham Farghal ; Ayoub, Haytham A. ; Ibrahim, Amina M.

Land snails are the most harmful pests in agricultural fields. Eobania vermiculata is a widespread snail species that causes massive damage to all agricultural crops. Thus, the molluscicidal activity of calcium borate nanoparticles (CB-NPs) against Eobania vermiculata was evaluated and compared with metaldehyde (Gastrotox® E 5% G). The amorphous phase of CB-NPs was obtained after thermal treatment at a low temperature (500 °C) which conformed by X-ray diffraction (XRD) analysis. CB-NPs are composed of aggregated nano-sheets with an average thickness of 54 nm which enhanced their molluscicidal activity. These nano-sheets displayed meso-porous network architecture with pore diameters of 13.65 nm, and a 9.46 m²/g specific surface area. CB-NPs and metaldehyde (Gastrotox® E 5% G) exhibited molluscicidal effects on Eobania vermiculata snails with median lethal concentrations LC₅₀ of 175.3 and 60.5 mg/l, respectively, after 72 h of exposure. The results also showed significant reductions of Eobania vermiculata snails hemocytes' mean total number, the levels of Testosterone (T) and Estrogen (E), alkaline phosphatase, acid phosphatase, albumin, and protein concentrations, succinate dehydrogenase, glucose, triglycerides and phospholipids levels, while significant increases in the phagocytic index and mortality index, both transaminases (ALT and AST) and glycogen phosphorylase concentration were observed after the exposure to LC₅₀ of CB-NPs or metaldehyde (Gastrotox® E 5% G) compared to the control group. Therefore, CB-NPs could be used as an alternative molluscicide for controlling Eobania vermiculata, but further studies are needed to assess their effects on non-target organisms.

03 Nov 2023·Blood transfusion = Trasfusione del sangue

Multi-component cord blood banking: a proof-of-concept international exercise.

Article

Author: Mallis, Panagiotis ; Rebulla, Paolo ; Michalopoulos, Efstathios ; Pello, Oscar M ; Gontica, Ioanna ; Samarkanova, Dinara ; Codinach, Margarita ; Gallerano, Pasquale ; Calvo, Javier ; Mykhailova, Larysa ; Montemurro, Tiziana ; Wynn, Liam ; Tancredi, Giuseppa ; Querol, Sergio

BACKGROUNDMost public cord blood (CB) banks currently discard more than 80% of umbilical CB units not suitable for hemopoietic stem cell transplant due to low stem cell count. Although CB platelets, plasma, and red blood cells have been used for experimental allogeneic applications in wound healing, corneal ulcer treatment, and neonatal transfusion, no standard procedures for their preparation have been defined internationally.MATERIALS AND METHODSA network of 12 public CB banks in Spain, Italy, Greece, the UK, and Singapore developed a protocol to validate a procedure for the routine production of CB platelet concentrate (CB-PC), CB platelet-poor plasma (CB-PPP), and CB leukoreduced red blood cells (CB-LR-RBC) using locally available equipment and the commercial BioNest ABC and EF medical devices. CB units with >50 mL volume (excluding anticoagulant) and ≥150×10⁹/L platelets were double centrifuged to obtain CB-PC, CB-PPP, and CB-RBC. The CB-RBC were diluted with saline-adenine-glucose-mannitol (SAGM), leukoreduced by filtration, stored at 2-6°C, and tested for hemolysis and potassium (K+) release over 15 days, with gamma irradiation performed on day 14. A set of acceptance criteria was pre-defined. This was for CB-PC: volume ≥5 mL and platelet count 800-1,200×10⁹/L; for CB-PPP: platelet count <50×10⁹/L; and for CB-LR-RBC: volume ≥20 mL, hematocrit 55-65%, residual leukocytes <0.2×10⁶/unit, and hemolysis ≤0.8%.RESULTSEight CB banks completed the validation exercise. Compliance with acceptance criteria was 99% for minimum volume and 86.1% for platelet count in CB-PC, and 90% for platelet count in CB-PPP. Compliance in CB-LR-RBC was 85.7% for minimum volume, 98.9% for residual leukocytes, and 90% for hematocrit. Compliance for hemolysis ≤0.8% decreased from 89.0 to 63.2% from day 0 to 15. K+ release increased from 3.0±1.8 to 25.0±7.0 mmol/L from day 0 to 15, respectively.DISCUSSIONThe MultiCord12 protocol was a useful tool to develop preliminary standardization of CB-PC, CB-PPP, and CB-LR-RBC.

100 Deals associated with CB-PURE(Vitti Labs LLC.)

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Autistic Disorder	IND Application	US	Vitti Labs LLC.	30 Nov 2022
Rheumatoid Arthritis	Preclinical	US	Vitti Labs LLC.	30 Nov 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

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


No Data

CB-PURE(Vitti Labs LLC.)

Overview

Basic Info

Related

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation