HPR63 Plasma Last Liter Economics and Its Impact on the Costs of Major Human Polyvalent Immunoglobulins in Europe
Human polyvalent immunoglobulins (Igs) are plasma derivatives that treat primary immunodeficiencies for which there are no alternatives. Unlike small molecule pharmaceutical products, the cost of plasma derivatives increases with the volume manufactured. Increasing demand for Igs results in high collection and fractionation costs and reduces the viability for manufacturers to increase production volumes. This research aims to explore the cost evolution of plasma in Europe and to demonstrate how the increasing Ig demand has driven costs. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: J. Galduf, M. Toumi, S. Neudoerfer, J. Sears, L. Marlow, J. Torres Ames Source Type: research

Challenges for gene editing in common variable immunodeficiency disorders: Current and future prospects
Clin Immunol. 2023 Nov 25:109854. doi: 10.1016/j.clim.2023.109854. Online ahead of print.ABSTRACTThe original CRISPR Cas9 gene editing system and subsequent innovations offers unprecedented opportunities to correct severe genetic defects including those causing Primary Immunodeficiencies (PIDs). Common Variable Immunodeficiency Disorders (CVID) are the most frequent symptomatic PID in adults and children. Unlike many other PIDs, patients meeting CVID criteria do not have a definable genetic defect and cannot be considered to have an inborn error of immunity (IEI). Patients with a CVID phenotype carrying a causative mutatio...
Source: Clinical Immunology - November 28, 2023 Category: Allergy & Immunology Authors: Rohan Ameratunga Euphemia Leung See-Tarn Woon Edward Lea Caroline Allan Lydia Chan Hilary Longhurst Richard Steele Russell Snell Klaus Lehnert Source Type: research

Challenges for gene editing in common variable immunodeficiency disorders: Current and future prospects
Clin Immunol. 2023 Nov 25:109854. doi: 10.1016/j.clim.2023.109854. Online ahead of print.ABSTRACTThe original CRISPR Cas9 gene editing system and subsequent innovations offers unprecedented opportunities to correct severe genetic defects including those causing Primary Immunodeficiencies (PIDs). Common Variable Immunodeficiency Disorders (CVID) are the most frequent symptomatic PID in adults and children. Unlike many other PIDs, patients meeting CVID criteria do not have a definable genetic defect and cannot be considered to have an inborn error of immunity (IEI). Patients with a CVID phenotype carrying a causative mutatio...
Source: Clinical Immunology - November 28, 2023 Category: Allergy & Immunology Authors: Rohan Ameratunga Euphemia Leung See-Tarn Woon Edward Lea Caroline Allan Lydia Chan Hilary Longhurst Richard Steele Russell Snell Klaus Lehnert Source Type: research

A novel tumor purity and immune infiltration-related model for predicting distant metastasis-free survival in prostate cancer
ConclusionsDynamic changes in the immune microenvironment associated with tumor purity could correlate with a poor DMFS of low-purity PCa. The TPS can predict the DMFS of PCa. In addition, prostate cancer metastases may be related to immunosuppression caused by a disorder of the immune microenvironment. (Source: European Journal of Medical Research)
Source: European Journal of Medical Research - November 28, 2023 Category: Research Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: Journal of Korean Medical Science - November 21, 2023 Category: Biomedical Science Authors: Joon Kee Lee Arum Oh Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: J Korean Med Sci - November 21, 2023 Category: General Medicine Authors: Joon Kee Lee Arum Oh Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: Anal Sci - November 21, 2023 Category: Chemistry Authors: Joon Kee Lee Arum Oh Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: Journal of Korean Medical Science - November 21, 2023 Category: Biomedical Science Authors: Joon Kee Lee Arum Oh Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: J Korean Med Sci - November 21, 2023 Category: General Medicine Authors: Joon Kee Lee Arum Oh Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: Journal of Korean Medical Science - November 21, 2023 Category: Biomedical Science Authors: Joon Kee Lee Arum Oh Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: J Korean Med Sci - November 21, 2023 Category: General Medicine Authors: Joon Kee Lee Arum Oh Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: Journal of Korean Medical Science - November 21, 2023 Category: Biomedical Science Authors: Joon Kee Lee Arum Oh Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: J Korean Med Sci - November 21, 2023 Category: General Medicine Authors: Joon Kee Lee Arum Oh Source Type: research

Combined Malonic and Methylmalonic Aciduria Diagnosed by Recurrent and Severe Infections Mimicking a Primary Immunodeficiency Disease: A Case Report
J Korean Med Sci. 2023 Nov 20;38(45):e387. doi: 10.3346/jkms.2023.38.e387.ABSTRACTCombined malonic and methylmalonic aciduria is a rare genetic disorder caused by ACSF3 biallelic variants that results in impaired protein and fat metabolism and the accumulation of malonic and methylmalonic acids. A 52-day-old infant with a fever and a history of possible meningitis during the neonatal period was hospitalized. Multiple lesions of necrotizing lymphadenitis with abscesses in the left inguinal area were treated by incision and drainage along with appropriate antibiotic therapy, which revealed a methicillin-resistant Staphylococ...
Source: Journal of Korean Medical Science - November 21, 2023 Category: Biomedical Science Authors: Joon Kee Lee Arum Oh Source Type: research