Multiple sclerosis and related disorders · 2025
Outcomes of autologous stem cell transplantation in patients with multiple sclerosis: A United Arab Emirates experience.
СтатьяНазвание и аннотация: Перевод в очереди
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Mir Ruqqia, Al-Kaabi Fatima, Brylev Lev, Abu-Haleeqa Mohammed, Dennison David, Handgretinger Rupert, Burt Richard, Bencomo-Hernandez Antonio Alfonso, AlKaram Maysoon, Ventura-Carmenate Yendry, Castillo-Aleman Yandy Marx, Afifi Yara, Ali Ruaa Ballal Mousa, Ali Sheima, Muhieddin Aseel, Al-Saadawi Nameer, El-Najjar Inas
Аннотация
BACKGROUND: Autologous hematopoietic stem-cell transplantation (AHSCT) is used for highly active, treatment-refractory multiple sclerosis (MS), but outcomes from the United Arab Emirates (UAE) are unreported. We evaluated safety, feasibility, and early effects on disability and inflammatory activity.
METHODS: Single-center retrospective series at Yas Clinic Khalifa City, Abu Dhabi (Jan 2022-Jan 2025). Adults (18-60) with EDSS ≥3.0 and persistent inflammatory activity despite high-efficacy DMT or active SPMS underwent mobilization with cyclophosphamide+G-CSF, conditioning with high-dose cyclophosphamide+ATG, and reinfusion of ≥2 × 10^6 CD34⁺ cells/kg.
PRIMARY OUTCOME: EDSS change at 3, 6, and 12 months; secondary outcomes: clinical relapses through 12 months and MRI activity at follow-up.
RESULTS: Ten patients (7 women; mean age 28.9 years) were treated. Mean CD34⁺ dose was 7.43 × 10^6 cells/kg. Neutrophil engraftment occurred at a median of 9.5 days; 8/10 had transient CMV/EBV reactivation; no treatment-related mortality occurred. Median EDSS was 5.5 at baseline, 5.5 at 3 months, 5.5 at 6 months, and 4.5 among five patients at 12 months. EDSS improved in 1/10 at 3 months, 2/10 at 6 months, and 3/5 at 12 months; the remainder were stable and no patient worsened during available follow-up. Median relapses were 0 in the first 6 and 12 months; no new gadolinium-enhancing lesions were detected at any follow-up visit.
CONCLUSIONS: In this first UAE experience, AHSCT was feasible and safe and produced early disability stabilization with progressive improvement among evaluable patients by 12 months, and complete suppression of focal inflammatory activity during follow-up. These data support integrating AHSCT into regional MS care while larger prospective studies define long-term benefit.
METHODS: Single-center retrospective series at Yas Clinic Khalifa City, Abu Dhabi (Jan 2022-Jan 2025). Adults (18-60) with EDSS ≥3.0 and persistent inflammatory activity despite high-efficacy DMT or active SPMS underwent mobilization with cyclophosphamide+G-CSF, conditioning with high-dose cyclophosphamide+ATG, and reinfusion of ≥2 × 10^6 CD34⁺ cells/kg.
PRIMARY OUTCOME: EDSS change at 3, 6, and 12 months; secondary outcomes: clinical relapses through 12 months and MRI activity at follow-up.
RESULTS: Ten patients (7 women; mean age 28.9 years) were treated. Mean CD34⁺ dose was 7.43 × 10^6 cells/kg. Neutrophil engraftment occurred at a median of 9.5 days; 8/10 had transient CMV/EBV reactivation; no treatment-related mortality occurred. Median EDSS was 5.5 at baseline, 5.5 at 3 months, 5.5 at 6 months, and 4.5 among five patients at 12 months. EDSS improved in 1/10 at 3 months, 2/10 at 6 months, and 3/5 at 12 months; the remainder were stable and no patient worsened during available follow-up. Median relapses were 0 in the first 6 and 12 months; no new gadolinium-enhancing lesions were detected at any follow-up visit.
CONCLUSIONS: In this first UAE experience, AHSCT was feasible and safe and produced early disability stabilization with progressive improvement among evaluable patients by 12 months, and complete suppression of focal inflammatory activity during follow-up. These data support integrating AHSCT into regional MS care while larger prospective studies define long-term benefit.