Study register · detailPhase-2-Pilotstudie (einarmig, open-label) · Cancer · 2025
Pilot Study of Cannabidiol for Treatment of Aromatase Inhibitor-Associated Musculoskeletal Symptoms in Breast Cancer.
Fleege et al.·Cancer MedicineImpact 3.3
MixedGRADEModerate5 citations
Samplen = 39 Pat.
Duration15 weeks
EndpointWorst Pain
Blindingoffen
DesignPhase-2-Pilotstudie (einarmig, open-label)
Cannabinoidcbd
Max. dose200.0 mg
Routeoral
”Key finding
43,6% of patients reached the primary endpoint definition (≥2-point reduction in pain); across all patients, worst pain improved by an average of 1,95 points over 15 weeks, and by 2,36 points among study completers; 28% of patients discontinued due to toxicity or preference.
Summary
Phase 2 pilot study (n=39) of CBD (Epidiolex, 100 mg BID) in breast cancer patients (stages 0–3) with aromatase inhibitor-associated musculoskeletal symptoms (AIMSS). 43,6 % (95 % CI [28 %, 60 %]) reached the primary endpoint (≥2-point reduction in worst-pain score). Worst pain improved by 0,13 points/week (p<0,001); mean reduction at study completion (n=28) 2,36 points (95 % CI [-3,22; -1,49]). CBD was safe and well tolerated.
P
PopulationWomen with hormone receptor-positive breast cancer stage 0–3 with aromatase inhibitor-associated musculoskeletal symptoms (AIMSS), n=39
I
InterventionCBD (Epidiolex), titrated over 4 weeks to 100 mg twice daily, total duration 15 weeks
O
Outcome17 of 39 patients (43,6 %, 95%-CI [28 %, 60 %]) reached the primary endpoint; average improvement in worst-pain score 1,95 points after 15 weeks (p<0,001)
Confidence in the evidence
Very lowLowModerateHigh
Moderate
The third of four GRADE levels, the effect estimate is probably reliable.
Downgraded for
Imprecision
Quality profile
Sample size★★★★★
BlindingOpen-label
Effect sizeMixed
Citations / year★★★★★
Authors
Fleege NMG, Miller EA, Kidwell KM, Zacharias ZR, Houtman J, Scheu K, Kemmer K, Boehnke KF, Henry NL
Aromatase inhibitor (AI) therapy reduces breast cancer recurrence risk. However, some patients stop treatment early because of AI-associated musculoskeletal symptoms (AIMSS). AIMSS is due in part to systemic inflammation. Cannabidiol (CBD) has anti-nociceptive and anti-inflammatory properties, making it a potential treatment option for AIMSS. Women with stage 0-3 hormone receptor-positive breast cancer experiencing AIMSS enrolled in this phase 2 clinical trial. Patients received CBD (Epidiolex), titrated over 4 weeks to 100 mg BID, for a total of 15 weeks. Patient-reported outcomes were collected serially. The primary endpoint was the number of patients with at least a 2-point reduction in worst pain from baseline to 15 weeks. Statistical analysis was completed using paired t-tests and linear mixed models. Of 39 eligible patients, 28 completed protocol-directed study treatment. Eleven discontinued treatment due to toxicity (n=5) or per patient preference (n=6). Seventeen of 39 patients met the primary endpoint (43.6%, 95% CI [28%, 60%]). Worst pain improved 0.13 per week of treatment (p<0.001) for all patients; average improvement in worst pain was 1.95 points at the end of 15 weeks. Of the 28 patients who completed the study, average reduction in worst pain was 2.36 points (95% CI [-3.22, -1.49]) between baseline and Week 15. Treatment with CBD was safe, tolerable, and associated with improvement in joint pain for a subset of patients. Additional studies are needed to further understand the impact of CBD on AIMSS and which patients are most likely to benefit from CBD treatment.