Reply to “Prophylactic Allogeneic Hematopoietic Stem Cell Therapy for CSF1R‐Related Leukoencephalopathy”

Abstract

Parkinson's disease (PD) is one of the most common progressive, systemic, and incurable neurodegenerative disorders with a high mortality rate and an increased risk of premature death when compared to the general population. 1 Actually, FIG 1. Hypothetical model for CSF1R-related leukoencephalopathy disease progression and proposed optimal timing for hematopoietic stem cell transplantation (HSCT) for patients with known CSF1R pathogenic variants. The gray shading corresponds with the optimal timing for HSCT; that is, darker shading correlates with better disease stages during which to consider HSCT. Years are based on published averages of symptom onset and disease duration. 4 Activities of daily living (ADL), mild cognitive impairment (MCI), mild behavioral impairment (MBI), and Sundal magnetic resonance imaging severity scale Total Score(SSS Tot ). 3 [Color figure can be viewed at wileyonlinelibrary.com]

The Phenotypic and Genotypic Spectrum of CSF1R‐Related Disorder in China

“…Because it has been revealed that motor-predominant patients responded better to hematopoietic stem cell transplantation (HSCT), which was the only potential therapeutic effects to stabilize disease progression and improve survival, 31,32 Chinese patients might have better response to HSCT from this prospective. Besides, autonomic involvement and peripheral neuropathy were rarely reported in patients from other regions, whereas in our cohort, 15.3% had autonomic symptoms and 34.4% were with peripheral EMG and/or CNV abnormality.…”

“…Similarly, during the course of CRD, the proportion of parkinsonism (76.2%) was also higher than that reported in the foreign population (60.7%). Because it has been revealed that motor‐predominant patients responded better to hematopoietic stem cell transplantation (HSCT), which was the only potential therapeutic effects to stabilize disease progression and improve survival, 31,32 Chinese patients might have better response to HSCT from this prospective.…”