What evidence exists for using perispinal etanercept for post-stroke symptoms?

Comment by InpharmD Researcher

Current evidence regarding perispinal etanercept for persistent stroke symptoms remains inconclusive, with a recent systematic review concluding that available data do not support routine clinical use. While a small randomized trial reported reductions in pain and improved shoulder flexion, and uncontrolled studies reported improvements across multiple neurological domains, the largest randomized, placebo-controlled trial found no significant improvement in quality of life or other efficacy outcomes (see Table 1). Additionally, an American Academy of Neurology practice advisory found insufficient evidence to support or refute efficacy and recommended counseling patients regarding unestablished effectiveness, potential adverse outcomes, and high cost. Although serious adverse events occurred at similar frequencies between groups in the larger randomized trial, shingles was reported in one participant in the smaller trial, and safety assessment remains limited by short follow-up and predominantly one- or two-dose exposure.

A literature search was conducted using PubMed and Google Scholar to identify clinical studies, systematic reviews, and guidelines evaluating the efficacy and safety of perispinal etanercept for post-stroke symptoms and functional recovery.

Background

A 2016 American Academy of Neurology (AAN) practice advisory evaluated the effectiveness, safety, and tolerability of etanercept for poststroke disability and identified two nonmasked, uncontrolled retrospective case series involving 3 and 617 patients with stroke; both were classified as Class IV evidence because of a high risk of bias. In the larger study, patients received posterior cervical perispinal injections of etanercept 25 mg followed by Trendelenburg positioning, with reported improvements across multiple functional domains immediately after treatment and maintenance of improvement in most patients assessed at 3 weeks. Neither study described adverse events following treatment; however, the advisory noted potentially serious adverse events reported with etanercept for other conditions and uncertainty regarding their applicability to time-limited perispinal administration. The authors concluded that evidence was insufficient to support or refute the efficacy of perispinal etanercept for poststroke disability and recommended counseling patients regarding uncertain effectiveness, potential adverse outcomes, and high cost. [1]

A 2025 systematic review evaluated the safety and effectiveness of perispinal etanercept for persistent post-stroke deficits, including five studies: two randomized controlled trials, one observational cohort, one case series, and one case report. Six databases were searched through September 2025, with two reviewers independently screening studies, extracting data, and assessing risk of bias; findings were synthesized qualitatively because differences in outcome measures and assessment times precluded quantitative pooling. A multicenter, double-blind randomized trial involving 126 participants found no significant improvement in the proportion achieving a ≥5-point increase in Short Form-36 scores with etanercept versus placebo (53% vs 58%; odds ratio [OR] 0.82; 95% confidence interval [CI] 0.40 to 1.67; p= 0.58). A smaller, double-blind randomized trial involving 22 participants reported reductions in pain scores and improved shoulder flexion following two 25-mg injections administered 14 days apart, with effects sustained through day 30. Uncontrolled studies reported improvements in motor function, cognition, speech, pain, and hemispatial neglect, but were assessed as having a high risk of bias; the larger randomized trial was rated as having low risk of bias, whereas the smaller trial had some concerns. Serious adverse events occurred at similar frequencies in the larger trial (2% with etanercept vs 3% with placebo), while one participant in the smaller trial developed shingles and was withdrawn. Safety assessment was limited by short follow-up and predominantly one- or two-dose exposure. The authors concluded that the overall evidence remains inconclusive and does not support routine clinical use, emphasizing the need for larger randomized trials with standardized outcomes and longer follow-up. [2]

A 2022 network meta-analysis evaluated pharmacotherapies for central post-stroke pain using 13 randomized controlled trials identified through searches of four databases from inception to March 30, 2022. Evidence for perispinal etanercept was derived from one parallel-group randomized trial involving 26 participants that compared etanercept 25 mg with 0.9% saline over a 2-week treatment period. Etanercept significantly reduced pain intensity compared with placebo (standardized mean difference [MD] −0.92; 95% CI −1.80 to −0.03). The review reported shingles in 1 of 13 etanercept recipients and no serious adverse events across the included pharmacotherapies; adverse events were not quantitatively pooled because reporting varied among trials. Functional status and quality of life were not assessed in the meta-analysis, and evidence of benefit beyond 3 months was lacking. The authors recommended further randomized trials of etanercept and additional evaluation of long-term effectiveness and safety. [3]

Background References: [1] Gronseth GS, Messé SR. Practice advisory: etanercept for poststroke disability: report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology. Neurology. 2016;86(23):2208-2211.
[2] Sanghi M, Khanijoun N, Hamza N. Evaluating the safety and effectiveness of perispinal etanercept for post-stroke recovery: a systematic review. Cureus. 2025;17(11):e96707. doi:10.7759/cureus.96707.
[3] Bo Z, Jian Y, Yan L, et al. Pharmacotherapies for central post-stroke pain: a systematic review and network meta-analysis. Oxid Med Cell Longev. 2022;2022:3511385. doi:10.1155/2022/3511385.
Literature Review

A search of the published medical literature revealed 5 studies investigating the researchable question:

What evidence exists for using perispinal etanercept for post-stroke symptoms?

Level of evidence

C - Multiple studies with limitations or conflicting results  Read more→



Please see Tables 1-5 for your response.


 

Safety and Efficacy of Perispinal Etanercept for Chronic Stroke
Design

Randomized, double-blind, placebo-controlled, parallel group trial

N=126

Objective To evaluate the safety and efficacy of perispinal etanercept in patients with chronic stroke
Study Groups

Etanercept (n=63)

Placebo (n=63)

Inclusion Criteria Patients aged 18-70 years with a stroke 1-15 years prior, mRS score of 2-5, and reduced quality of life as assessed by SF-36
Exclusion Criteria Contraindications to etanercept, certain infectious diseases, immunosuppressive agent use, severe heart conditions, malignancy history, or recent botulinum toxin injection
Methods Participants received a single dose of etanercept 25 mg or placebo via subcutaneous injection in the posterior cervical region. Follow-up assessments were conducted at 28 and 56 days post-injection
Duration November 2020 to September 2023
Outcome Measures

Primary: Improvement of 5 points or more on the SF-36v2 at day 28

Secondary: Improvement of 5 points or more on the SF-36v2 at day 56

Baseline Characteristics   Placebo (n=63) Etanercept (n=63)
Age at enrollment, y, median (IQR) 55.00 (46.00–60.00) 54.00 (45.00–61.00)
Sex, female, n (%) 25 (40) 23 (37)
Stroke type - Ischemic stroke, n (%) 38 (60) 47 (75)
Hypertension, n (%) 27 (43) 28 (47)
Baseline NIHSS score, median (IQR) 6.00 (3.00–9.00) 5.00 (3.00–8.00)
Results

In the prespecified intention-to-treat analysis, etanercept did not significantly improve SF-36v2 scores compared with placebo at day 56 (53% vs 58% achieved ≥5-point improvement; adjusted OR 0.82; 95% CI 0.40–1.67). No differences were observed in the total, physical, or mental component scores. Similarly, improvement rates were comparable between participants receiving two doses of etanercept (54%), one dose (66%), or two doses of placebo (58%).

Adverse Events The proportion of serious adverse events was similar in both treatment arms. Five serious adverse events were reported through day 56: two seizures (one participant receiving placebo), renal carcinoma with pancreatic and brain metastases (etanercept group), a femoral neck fracture (placebo group), and malaise with possible aspiration (etanercept followed by placebo).
Study Author Conclusions Perispinal etanercept was safe, but no evidence of improvement in quality of life and other efficacy outcomes compared with placebo/
Critique The study was well-designed with a randomized, double-blind, placebo-controlled approach, but it was limited by early termination due to funding issues, which may have affected the power to detect small treatment effects. The lack of standardized rehabilitation and potential biases due to proxy reporting are additional limitations.
Table 1 References:
[4] Thijs VN, Cloud GC, Gilchrist N, et al. Safety and Efficacy of Perispinal Etanercept for Chronic Stroke: A Randomized Clinical Trial. Neurology. 2025;105(6):e213981. doi:10.1212/WNL.0000000000213981

Phase I/II Parallel Double-Blind Randomized Controlled Clinical Trial of Perispinal Etanercept for Chronic Stroke: Improved Mobility and Pain Alleviation

Design

Phase I/II parallel double-blind randomized controlled clinical trial

N= 22

Objective

To test perispinal etanercept for chronic post-stroke pain and assess its effects on mobility and pain alleviation

Study Groups

Etanercept (n= 10)

Control (n= 12)

Inclusion Criteria

Aged between 30 and 80 years; stroke occurring at least 6 months and not more than 15 years prior; chronic neurological impairment following an ischemic stroke in the territory of the middle cerebral arteries or basal ganglia form of intracerebral hemorrhage; constant daily pain post-stroke with moderate-to-severe intensity

Exclusion Criteria

Not specified in the provided text

Methods

Participants were randomized to perispinal etanercept 25 mg or saline 1.8 mL on days 1 and 14, with participants, investigators, and assessors blinded to allocation. Injections were administered subcutaneously at C6–C7 or C7–T1, followed by 4 minutes of 45° Trendelenburg positioning. Regular medications remained unchanged, and participants maintained pain diaries from day −7 through day 30. Pain was assessed before and 30–60 minutes after each treatment and by telephone on day 30; additional assessments evaluated shoulder flexion, cognition, fatigue, depression, functional performance, and sensory thresholds. Analyses included 22 completers in their assigned groups; the trial stopped early after statistically significant interim findings.

Duration

November 2016 through March 2019

Outcome Measures

Primary: Differences in daily pain levels between groups

Secondary: Improved shoulder flexion, spasticity, cognition, executive function, hemispatial neglect, and post-stroke depression

Baseline Characteristics

 

Etanercept (n= 10) Saline control (n= 12)

Age, years

57.3 (4.95) 61.65 (8.66)

Weight, kg

77.74 (5.73) 85.85 (4.86)

Female

5 (50%) 5 (41.7%)

Years since stroke

4.18 (0.72) 4.98 (1.15)

Average daily pain (vNPRS-FPS)

68 (3.35) 60.42 (485)

Worst daily pain

82.5 (3.96) 74.58 (3.45)

Active Shoulder Flexion/ROM

55 (15.69) 84.11 (13.26)

Passive Shoulder Flexion/ROM

103 (10.25) 116.11 (12.35)

MOCA

21.8 (2.33) 25.5 (0.77)

BDI

17.6 (3.32) 20.8 (3.36)

FAS

31.4 (1.54) 34.8 (1.62)

STS

19.5 (2.7) 24.4 (3.5)

Abbreviations: MOCA, Montreal Cognitive Assessment; BDI, Beck’s Depression Inventory; FAS; Fatigue Assessment Score; STS; Five times Sit-To-Stand.

Results

 

Etanercept (n= 10) Control (n= 12) p-value

Average daily pain reduction (0-100 scale)

24 ± 9 2.5 0.04

Worst daily pain reduction (0-100 scale)

19.5 ± 6 2.5 0.023

Improvement in shoulder flexion (degrees)

55 ± 12 0 0.011

Among 22 completers (etanercept, n= 10; saline, n= 12), mean average daily pain scores decreased from 68.0 to 44.1 with etanercept versus 60.42 to 57.50 with saline by day 30; the between-group comparison of changes favored etanercept (Hodges–Lehmann estimate, 15 points; 95% confidence interval [CI] 0 to 40; p= 0.040).

Mean worst daily pain scores decreased from 82.5 to 63.0 versus 74.58 to 72.08, respectively (Hodges–Lehmann estimate, 15 points; 95% CI 5 to 30; p= 0.023).

Three of 10 etanercept recipients experienced complete or almost complete pain resolution within approximately 30 minutes after the first injection, whereas four had no or limited pain improvement.

Mean active shoulder flexion increased by 55° (SE, 12°) after the second etanercept injection, with a significant between-group difference in change (p= 0.011); nine of 10 etanercept recipients improved, including three who regained 180° flexion.

Between-group differences in cognition, depression, fatigue, sit-to-stand performance, and thermal detection or pain thresholds were not statistically significant.

One etanercept recipient developed shingles after the first injection and discontinued participation; no serious adverse events were reported.

Adverse Events

No serious adverse events recorded. One patient developed shingles after treatment, considered a treatment-emergent adverse event of special interest.

Study Author Conclusions

Perispinal etanercept can provide significant and ongoing benefits for the chronic post-stroke management of pain and greater shoulder flexion by the paretic arm. Effects are rapid and highly significant, supporting direct action on brain function.

Critique

Randomization, concealed allocation, double blinding, and a saline procedure control strengthen the comparison, but the small completer-only analysis, exclusions after randomization, and early stopping for benefit may overestimate treatment effects. Follow-up through day 30 limits assessment of durability and safety, while eligibility changes, addition of outcomes after trial commencement, and inconsistent statistical reporting complicate interpretation beyond the observed pain and shoulder-flexion findings.

Table 2 References:
[5] Ralph SJ, Weissenberger A, Bonev V, et al. Phase I/II parallel double-blind randomized controlled clinical trial of perispinal etanercept for chronic stroke: improved mobility and pain alleviation. Expert Opin Investig Drugs. 2020;29(3):311-326. doi:10.1080/13543784.2020.1709822
Selective TNF Inhibition for Chronic Stroke and Traumatic Brain Injury: An Observational Study Involving 629 Consecutive Patients Treated with Perispinal Etanercept
Design

Observational study

N= 629

Objective To systematically examine the clinical response following perispinal administration of etanercept in a cohort of patients with chronic neurological dysfunction after stroke and TBI
Study Groups

Stroke patients (n= 617)

TBI patients (n= 12)

Inclusion Criteria Patients with chronic neurological dysfunction following stroke or TBI who were treated with perispinal etanercept from November 1, 2010 to July 14, 2012
Exclusion Criteria Not specified
Methods Patients were treated with perispinal etanercept (PSE) 25 mg. Assessments were performed immediately following the first dose, and at 1 week and 3 weeks after the first dose. Quantitative data included time to walk 20 m, hand grip strength, visual analogue score for pain, Montreal Cognitive Assessment, and controlled oral word association test. Qualitative assessments included motor impairment, spasticity, cognitive impairment, and other clinical symptoms
Duration November 1, 2010 to July 14, 2012
Outcome Measures

Clinical improvement in motor function, spasticity, cognition, psychological/behavioral function, aphasia, and pain

Baseline Characteristics   Stroke patients (n= 617) TBI patients (n= 12)
Age, years (mean ± SD) 65.8 ± 13.15 34.7 ± 13.81
Months since event (mean ± SD) 42.0 ± 57.84 115.2 ± 160.22
Male 372 (60.3%) 6 (50.0%)
Female 245 (39.7%) 6 (50.0%)
Results   Stroke patients TBI patients
Improvement in motor function Yes Yes
Improvement in spasticity Yes Yes
Improvement in cognition Yes Yes
Improvement in psychological/behavioral function Yes Yes
Improvement in pain Yes Yes
Adverse Events Not specified
Study Author Conclusions Excess TNF contributes to chronic neurological, neuropsychiatric, and clinical impairment after stroke and TBI. Perispinal administration of etanercept produces clinical improvement in patients with chronic neurological dysfunction following stroke and TBI. The therapeutic window extends beyond a decade after stroke and TBI.
Critique The study provides promising evidence of clinical improvement in chronic neurological dysfunction after stroke and TBI with perispinal etanercept. However, the open-label design, lack of a control group, and potential for placebo effects limit the strength of the conclusions. Randomized controlled trials are necessary to confirm these findings and establish the safety and efficacy of the treatment.
Table 3 References:
[6] Tobinick E, Kim NM, Reyzin G, Rodriguez-Romanacce H, DePuy V. Selective TNF inhibition for chronic stroke and traumatic brain injury: an observational study involving 629 consecutive patients treated with perispinal etanercept. CNS Drugs. 2012;26(12):1051-1070. doi:10.1007/s40263-012-0013-2

Rapid Improvement of Chronic Stroke Deficits After Perispinal Etanercept: Three Consecutive Cases

Design

Case series

Case presentation 1

A 61-year-old man presented 36 months after a left middle cerebral artery stroke with persistent right hemiparesis, hemianaesthesia, upper-extremity spasticity, severe expressive aphasia, and impaired gait, cognition, and daily functioning despite previous rehabilitation.

He received perispinal etanercept 25 mg followed by 5 minutes of Trendelenburg positioning. Speech became more distinct within 2 minutes, followed by improvements in right-arm mobility, sensation, and spasticity; at 2 hours, his outward/return 20-metre walking times decreased from 19.8/23.0 seconds to 10.1/11.6 seconds. Subsequent improvements included the ability to dial telephone numbers, tell time, type a sentence, and run.

At day 22, his Mini-Mental State Examination (MMSE) score increased from 26/30 to 28/30, Montreal Cognitive Assessment (MoCA) score from 23/30 to 27/30, and activities of daily living inventory score from 61/78 to 65/78. A second perispinal etanercept dose administered that day was followed by improved speech articulation and the return of voluntary right-wrist dorsiflexion. Clinical improvement persisted at 8 weeks, with no adverse effects reported.

Case presentation 2

A 49-year-old man presented 35 months after a right medullary stroke with persistent left hemiparesis, hypoaesthesia, painful left upper-extremity paraesthesia, and impaired balance and gait requiring a walker; his neurological deficits had remained stable for at least 1 year.

He received perispinal etanercept 25 mg followed by 5 minutes of Trendelenburg positioning. Standing balance improved at 9 minutes, and outward/return 20-metre walking times using a standard walker decreased from 116/123 seconds before treatment to 80/81 seconds at 30 minutes and 66/71 seconds at day 10.

At day 17, walking times were 73/70 seconds, with a longer stride, more fluid gait, and reported reduction in the effort required for walking and incorporating his left arm into daily activities. A second 25 mg dose was administered on day 24; 10 minutes afterward, walking times were 63/63 seconds. At 1 month, improvements in walking and subtle improvements in left upper-extremity motor control were maintained, with no adverse effects noted.

Case presentation 3

A 58-year-old man presented 13 months after a right middle cerebral artery stroke with persistent left hemiparesis, hypoaesthesia, upper-extremity spasticity, impaired left-hand dexterity and spatial perception, left arm/hand pain, and difficulties with gait, balance, and daily activities; he reported no neurological improvement for at least 6 months.

He received perispinal etanercept 25 mg followed by 5 minutes of Trendelenburg positioning. Left facial droop improved beginning at 7 minutes, and left-hand tapping speed increased from 2.8 Hz to 5.5 Hz at 8–10 minutes. At 11–15 minutes, sensation and spatial perception improved, left/right grip strength increased from 32/36 to 36/40, and firm left-hand gripping no longer produced pain; measurement units for grip strength were not reported. Grip strength reached 40/40 at day 7, and outward/return 20-metre walking times decreased from 13/14 seconds at baseline to 10/10 seconds at day 13.

At day 13, MMSE remained 26/30, while MoCA increased from 23/30 to 29/30; improved abilities included manipulating buttons, buckling a belt, and handling playing cards. Left-hand improvement began diminishing at day 20, and a second 25 mg dose on day 26 was followed within 30 minutes by easier shoe lacing and improvement in facial droop. At 1 month, clinical improvement from baseline continued, with no adverse effects noted.

Study Author Conclusions

Open-label administration of perispinal etanercept resulted in rapid neurological improvement in three consecutive patients with chronic neurological dysfunction due to strokes occurring 13–36 months earlier. These results suggest that stroke may result in chronic TNF-mediated pathophysiology that may be amenable to therapeutic intervention long after the acute event. Randomized clinical trials of perispinal etanercept for selected patients with chronic neurological dysfunction following stroke are indicated.

Table 4 References:
[7] Tobinick E. Rapid improvement of chronic stroke deficits after perispinal etanercept: three consecutive cases. CNS Drugs. 2011;25(2):145-155. doi:10.2165/11588400-000000000-00000

Immediate Resolution of Hemispatial Neglect and Central Post-Stroke Pain After Perispinal Etanercept: Case Report

Design

Case report

Case presentation

A 59-year-old man presented 16 years after a right middle cerebral artery stroke complicated by malignant cerebral edema and coma requiring decompressive craniectomy and subsequent cranioplasty. Persistent deficits included left hemiparesis, hemispatial neglect, visuospatial dysfunction, sensory loss, cold allodynia, spasticity, cognitive dysfunction, fatigue, and constant left-sided central post-stroke pain involving the shoulder, forearm, and lower extremity. He required a left ankle-foot orthosis and single-point cane for ambulation.

Before treatment, his Montreal Cognitive Assessment score was 19/30, Fatigue Assessment Scale score was 24/50, pain severity was 5/10, 20-metre walking time was 58 seconds, and Five Times Sit-to-Stand time was 27 seconds with assistance from his right arm. Following informed consent, he received a single perispinal etanercept 25 mg injection followed by Trendelenburg positioning. Within 10 minutes, he reported complete resolution of his chronic left-sided pain; within 30 minutes, he reported reduced spasticity and improvements in gait, balance, turning left, and perception of his left foot’s position. Repeat clock drawing at 30 minutes no longer demonstrated left hemispatial neglect.

At telephone follow-up 17 days later, he reported maintained neurological improvements, absence of pain and allodynia, and improved peripheral vision. His Fatigue Assessment Scale score decreased to 17/50 at both 1 and 2 months. At telephone follow-up 11 months after treatment, he reported sustained neurological improvement and no recurrence of his previously constant post-stroke pain. Adverse events were not reported.

Study Author Conclusions

The striking pattern of immediate improvement in clock drawing documented in this patient with chronic, previously intractable, central post-stroke pain, chronic post-stroke fatigue, and left hemispatial neglect is most consistent with rapid restoration of brain functional connectivity. This evidence suggests hemispatial neglect may be caused by TNF-mediated network dysfunction and impairment in functional connectivity that remains reversible, even years after stroke. This evidence also suggests that central post-stroke pain may be mediated by TNF.

Table 5 References:
[8] Tobinick E. Immediate Resolution of Hemispatial Neglect and Central Post-Stroke Pain After Perispinal Etanercept: Case Report. Clin Drug Investig. 2020;40(1):93-97. doi:10.1007/s40261-019-00864-8