Abstract
BACKGROUND: Autologous hematopoietic stem cell transplantation (aHSCT) is an emerging treatment option for patients with relapsing-remitting multiple sclerosis (RRMS) or secondary progressive multiple sclerosis (SPMS) who exhibit ongoing disease activity despite disease-modifying therapy (DMT). Limited access to high-efficacy DMTs in the Philippines makes aHSCT an important escalation strategy. This study describes the clinical characteristics, transplant course, and short-term outcomes of Filipino patients with multiple sclerosis (MS) who underwent aHSCT at a single tertiary center. METHODOLOGY: We conducted a retrospective observational review of all patients with MS who underwent aHSCT as salvage or escalation therapy for ongoing disease activity despite prior DMTs at St. Luke's Medical Center from April 2023 to March 2025. Extracted data included demographics, MS phenotype, disease duration, baseline neurologic functional status, mobilization and conditioning regimens, transplant-related complications, magnetic resonance imaging (MRI) findings, post-transplant clinical outcomes, and No Evidence of Disease Activity (NEDA) status. Conditioning regimens included carmustine, cytarabine, etoposide, melphalan, and anti-thymocyte globulin (ATG) in five patients (83.3%) and cyclophosphamide-methylprednisolone-ATG in one patient (16.7%). Descriptive statistics were used to summarize outcomes. RESULTS: Six Filipino patients (median age: 44.5 years) were included. Five patients (83.3%) were female, and one (16.7%) was male. Five patients (83.3%) had SPMS, and one patient (16.7%) had RRMS. The mean disease duration was 14.3 years, and five patients (83.3%) had moderate to severe neurologic disability at baseline requiring ambulatory assistance. Rituximab was the most commonly used pre-aHSCT DMT. Febrile neutropenia occurred in five patients (83.3%), and one patient (16.7%) developed a central line-associated bloodstream infection; all patients recovered. During a mean follow-up of 17.2 months, no patient experienced clinical relapse or worsening functional disability, and none required resumption of DMTs. Two patients (33.3%) who underwent interval MRI demonstrated no new T2 or gadolinium-enhancing lesions. Four patients (66.7%) achieved NEDA-2, and two patients (33.3%) with interval MRI follow-up achieved NEDA-3. CONCLUSIONS: aHSCT was well tolerated and associated with short-term clinical stability in Filipino patients with active RRMS and SPMS. During follow-up, all patients remained relapse-free without evidence of confirmed disability progression. These findings support the role of aHSCT as a viable therapeutic option in settings with limited access to high-efficacy DMTs. Larger studies with longer follow-up are needed to evaluate the durability of remission.