J Neurol Res

Journal of Neurology Research, ISSN 1923-2845 print, 1923-2853 online, Open Access

Article copyright, the authors; Journal compilation copyright, J Neurol Res and Elmer Press Inc

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Case Report

Volume 16, Number 3, September 2026, pages 186-191

Autologous Hematopoietic Stem Cell Transplantation in Aquaporin-4 Immunoglobulin G–Positive Neuromyelitis Optica Spectrum Disorder After Satralizumab Failure

↓ Figure 1. Sagittal spinal MRI demonstrating disease evolution. (a) Extensive cervical myelitis at NMOSD onset. (b) Follow-up MRI performed approximately 18 months later showing a new longitudinally extensive thoracic spinal cord lesion, consistent with ongoing disease activity.

↓ Figure 2. Longitudinal clinical course, key events, and treatment exposure from 2017 to 2026. EDSS: Expanded Disability Status Scale; SLE: systemic lupus erythematosus; APS: antiphospholipid syndrome; NMOSD: neuromyelitis optica spectrum disorder; AQP4: aquaporin-4; AQP4-IgG: aquaporin-4 immunoglobulin G antibody; ITP: immune thrombocytopenia; aHSCT: autologous hematopoietic stem cell transplantation.

↓ **Table 1.** Summary of Reported Cases of aHSCT in NMOSD
References	Country	Sample size	Gender (female%)	Mean age (range)	Disease duration (years)	Comorbidities	Regimen	Serological status AQP4-IgG	Follow-up (years)	Prognosis
aHSCT: autologous hematopoietic stem cell transplantation; AQP4-IgG: aquaporin-4 immunoglobulin G; ATG: anti-thymocyte globulin; APS: antiphospholipid syndrome; BEAM: carmustine, etoposide, cytarabine, and melphalan; CTX: cyclophosphamide; G-CSF: granulocyte colony-stimulating factor; IBS: irritable bowel syndrome; SLE: systemic lupus erythematosus; NA: not available; NMOSD: neuromyelitis optica spectrum disorder; RTX: rituximab.
Peng et al, 2010 [17]	China	1	100%	23	2	No	CTX, G-CSF	Positive	1	Clinical remission after transplantation and no relapses over 12 months follow-up.
Greco et al, 2014 [20]	Italy	16	81.25%	37 (20–57)	NA	No	BEAM/ATG	Negative: 3; Positive: 10	5	Three cases remained progression- and treatment-free, while in 13 patients, anti-AQP-4Ab antibodies persisted, leading to relapse requiring further treatment.
Hoay & Ratnagopal, 2018 [18]	Singapore	3	66.7%	32	NA	No	CTX, fludarabine, ATG	Positive: 2 Not tested: 1	7.3 (4–13)	All three patients were neurologically stable, with no progression in their EDSS scores, or development of new lesions on their follow-up MRI.
Burt et al, 2019 [21]	USA	13	91.6%	42 (19–51)	7	One patient with SLE	BEAM/ATG	Negative: 1; Positive: 11	4 (2–5)	80% remain relapse free at year 5. Two patients remained AQP4-IgG-seropositive relapsed within 2 years of HSCT.
Carlisle et al, 2020 [19]	USA	1	100%	40	1	IBS, depression, and anxiety	BEAM/ATG	Positive	4	Mild relapse at 2-year follow-up, started eculizumab.
Bruton et al, 2021 [23]	Canada	3	66%	34 (28–39)	8.3 (3–13)	No	CTX/RTX/ATG	Positive: 3	7.5 (3–10)	Of the three patients, one remained relapse-free for 10 years and became AQP4-IgG seronegative, one relapsed 5 years after aHSCT while remaining AQP4-IgG seropositive and started rituximab at year 6, and one died during follow-up.
Vorasoot et al, 2025 [24]	USA	2	100%	65 (60–70)	30	No	Not mentioned	Positive	10	Both patients achieved AQP4-IgG seronegativity and relapsed 10 years after aHSCT.
Zertuche et al, 2026	Mexico	1	100%	32	3	SLE, APS, and hypothyroidism	CTX/RTX/ATG	Positive	2	Relapse free, remains AQP4 positive at 2-year follow-up.

↓ Table 1. Summary of Reported Cases of aHSCT in NMOSD

References

Country

Sample size

Gender (female%)

Mean age (range)

Disease duration (years)

Comorbidities

Regimen

Serological status AQP4-IgG

Follow-up (years)

Prognosis

aHSCT: autologous hematopoietic stem cell transplantation; AQP4-IgG: aquaporin-4 immunoglobulin G; ATG: anti-thymocyte globulin; APS: antiphospholipid syndrome; BEAM: carmustine, etoposide, cytarabine, and melphalan; CTX: cyclophosphamide; G-CSF: granulocyte colony-stimulating factor; IBS: irritable bowel syndrome; SLE: systemic lupus erythematosus; NA: not available; NMOSD: neuromyelitis optica spectrum disorder; RTX: rituximab.

Peng et al, 2010 [17]

China

100%

CTX, G-CSF

Positive

Clinical remission after transplantation and no relapses over 12 months follow-up.

Greco et al, 2014 [20]

Italy

81.25%

37 (20–57)

BEAM/ATG

Negative: 3; Positive: 10

Three cases remained progression- and treatment-free, while in 13 patients, anti-AQP-4Ab antibodies persisted, leading to relapse requiring further treatment.

Hoay & Ratnagopal, 2018 [18]

Singapore

66.7%

CTX, fludarabine, ATG

Positive: 2 Not tested: 1

7.3 (4–13)

All three patients were neurologically stable, with no progression in their EDSS scores, or development of new lesions on their follow-up MRI.

Burt et al, 2019 [21]

USA

91.6%

42 (19–51)

One patient with SLE

BEAM/ATG

Negative: 1; Positive: 11

4 (2–5)

80% remain relapse free at year 5. Two patients remained AQP4-IgG-seropositive relapsed within 2 years of HSCT.

Carlisle et al, 2020 [19]

USA

100%

IBS, depression, and anxiety

BEAM/ATG

Positive

Mild relapse at 2-year follow-up, started eculizumab.

Bruton et al, 2021 [23]

Canada

66%

34 (28–39)

8.3 (3–13)

CTX/RTX/ATG

Positive: 3

7.5 (3–10)

Of the three patients, one remained relapse-free for 10 years and became AQP4-IgG seronegative, one relapsed 5 years after aHSCT while remaining AQP4-IgG seropositive and started rituximab at year 6, and one died during follow-up.

Vorasoot et al, 2025 [24]

USA

100%

65 (60–70)

Not mentioned

Positive

Both patients achieved AQP4-IgG seronegativity and relapsed 10 years after aHSCT.

Zertuche et al, 2026

Mexico

100%

SLE, APS, and hypothyroidism

CTX/RTX/ATG

Positive

Relapse free, remains AQP4 positive at 2-year follow-up.