Pediatric congenital heart disease (CHD) pharmacotherapy faces three fundamental barriers: developmental pharmacokinetic complexity, anatomic-genetic heterogeneity, and evidence chain gaps. Traditional agents exhibit critical limitations: digoxin’s narrow therapeutic index (0.5–0.9 ng/mL) is exacerbated by ABCB1 mutations (toxicity risk increases 4.1-fold), furosemide efficacy declines by 35% in neonates due to NKCC2 immaturity, and β-blocker responses vary by CYP2D6 polymorphisms (poor metabolizers require 50–75% dose reduction). Novel strategies demonstrate transformative potential—CRISPR editing achieves 81% reversal of BMPR2-associated pulmonary vascular remodeling, metabolically matured cardiac organoids replicate adult myocardial energy metabolism for drug screening, and SGLT2 inhibitors activate triple mechanisms (calcium overload mitigation, mitophagy, fibrosis reversal). However, clinical translation requires overcoming developmental barriers: age-dependent enzyme expression (infant CYP2D6 = 30–60% adult activity), post-Fontan hepatotoxicity (bosentan trough concentrations elevates 1.8-fold), and AI model limitations (32% error in complex CHD). Future integration of placental transfer models, disease-specific organoids, and multi-omics mapping of FOXO/CRIM1 pathways will shift paradigms from symptom control to curative repair.

Mesenchymal stromal cells (MSCs) including mesenchymal stem cells to potentially differentiate into different tissue lineages widely exist in various tissues. In recent years, the clinical research and application of MSCs have become more extensive, but no standardized guidelines for the preparation and quality control of clinical-grade MSCs currently exist. To standardize the preparation and quality control of MSCs using the human umbilical cord, placenta, bone marrow, and adipose tissue as sample sources for the Chinese Association of Neurorestoratology (CANR; Preparatory) and the China Committee of International Association of Neurorestoratology (IANR-China Committee) member units, this standard is formulated following the T11/CSSCR 001-2017 General Requirements for Stem Cells, Good Manufacturing Practice Pharmaceutical Products (2010 Edition), Pharmacopoeia of the People's Republic of China (2015 Edition), Guiding Principles for Quality Control of Stem Cell Preparations and Preclinical Research (Trial), Code for Cell Banking Facility Quality Management, Sterile Drug Appendix to Pharmaceutical Production Quality Management Regulations, GMP Appendix — Cell Therapy Products (Draft for Comment), The International Society for Cellular Therapy position statement (2006), and Clinical Cell Therapy Guidelines for Neurorestoration (IANR/CANR 2017). Moreover, this standard includes donor evaluation, sample collection, cell preparation, cell inspection, packaging, labeling, transportation and storage, and quality control. It represents the minimum requirements for clinical-grade mesenchymal stromal cell culture and quality control. Moreover, it will be further optimized following the progress of preclinical and clinical research.