Hard - Clinical Trials and Emerging Therapies - PMR

Flashcards covering key clinical trials (SAPHYR, SEMAPHORE, REPLENISH, BACHELOR, GCAptAIN), specific agents like sarilumab and tocilizumab, and outcome measures for polymyalgia rheumatica (PMR) and giant cell arteritis (GCA).

SAPHYR phase 3 trial

The pivotal trial of sarilumab in relapsing (steroid-refractory) PMR, supporting its 2023 FDA approval.

Sustained remission (SAPHYR endpoint)

The primary endpoint of the SAPHYR trial at week $52$, consisting of symptom resolution by week $12$, sustained CRP normalization, no flare, and taper adherence.

Sarilumab sustained remission rate

In the SAPHYR trial, approximately $28.3\%$ of patients achieved sustained remission at $52$ weeks compared to $10.3\%$ in the placebo group ($p=0.0193$).

Median cumulative glucocorticoid dose (SAPHYR)

The amount of steroid exposure over $52$ weeks was reduced to $777\,mg$ with sarilumab compared to $2,044\,mg$ for placebo.

SEMAPHORE trial

A phase $3$ RCT evaluating IV tocilizumab in glucocorticoid-dependent PMR, where $67.3\%$ of the tocilizumab group met the composite primary endpoint at week $24$.

PMR-SPARE trial

A study that evaluated subcutaneous tocilizumab in new-onset PMR, using glucocorticoid-free remission at week $16$ as the primary endpoint.

REPLENISH phase 3 trial

A global phase $3$ trial reported in $2026$ evaluating secukinumab (an IL-17A inhibitor) in PMR, which met its primary and secondary endpoints.

Secukinumab (REPLENISH trial result)

In PMR patients, sustained remission at week $52$ was approximately $41.2\%$ for the $300\,mg$ dose and $40.6\%$ for the $150\,mg$ dose, versus $20.4\%$ for placebo.

GCAptAIN trial

A phase $3$ trial of secukinumab in giant cell arteritis (GCA) that missed its primary endpoint in $2026$, despite a positive phase $2$ result.

BACHELOR study

A proof-of-concept RCT that evaluated baricitinib in new-onset, glucocorticoid-naive PMR.

Baricitinib

An oral JAK1/2 inhibitor studied for early PMR in the BACHELOR trial.

Upadacitinib (Rinvoq)

The first oral JAK inhibitor FDA-approved for giant cell arteritis (GCA) in April $2025$, based on the SELECT-GCA trial.

Glucocorticoid-free remission

A trial endpoint that demonstrates a steroid-sparing effect by achieving remission without ongoing or rescue steroids.

Cumulative glucocorticoid dose (trial endpoint)

A surrogate measure used to capture both steroid-sparing efficacy and the safety/toxicity burden of treatment exposure.

OMERACT mandatory inner-core domains

Mandatory domains for PMR trials including laboratory markers of systemic inflammation, pain, stiffness, and physical function.

PMR Activity Score (PMR-AS)

A composite score calculated as CRP + patient pain VAS + physician global VAS + morning stiffness (min $\times 0.1$) + elevation of upper limbs (EUL $0-3$).

FDA-approved biologic for PMR (as of 2026)

Sarilumab ($2023$) is the only biologic with this specific approval, though secukinumab has been submitted to regulators following the REPLENISH trial.