Treatment of Active Moderate-to-Severe Graves' Orbitopathy with Exophthalmos and Diplopia

When Graves' orbitopathy (GO) is both moderate-to-severe in severity and immunologically active, it meets the threshold where intensive systemic treatment is warranted. This clinical scenario is defined by a combination of objective orbital findings and an active inflammatory state.

Defining Clinical Features

  • Clinical activity score (CAS) ≥ 3/7 — disease classified as active
  • Lid retraction ≥ 2 mm
  • Moderate or severe soft-tissue involvement
  • Exophthalmos ≥ 3 mm above normal for race and gender
  • Inconstant or constant diplopia

Treatment Approach — Partial Overview

For most patients in this category, first-line management involves a combination of intravenous immunosuppressive therapy with a concurrent oral immunomodulatory agent. For patients at the more severe end of the spectrum — including significant diplopia or markedly elevated exophthalmos — an alternative intravenous monotherapy regimen at a higher intensity may be preferred instead. Coexisting hyperthyroid state is controlled medically throughout the course of orbital treatment.

Specific agents, dosing, schedule, and decision criteria are detailed in the full protocol.

Treatment Goals

  • ≥ 2 mm reduction in lid aperture
  • ≥ 1 point reduction in the five-item CAS
  • ≥ 2 mm reduction in exophthalmos
  • ≥ 8° increase in eye muscle duction
  • Improvement in ≥ 2 features in one eye without deterioration in the other
  • Response assessed 3 months after the last intervention (changes at 6 months also considered)

References

DOI: 10.1530/EJE-21-0479

  • GO is defined as active if CAS is ≥ 3/7 (Table 2).
  • Lid retraction ≥ 2 mm; moderate or severe soft-tissue involvement; exophthalmos ≥ 3 mm above normal for race and gender; inconstant or constant diplopia.
  • Patients without sight-threatening GO whose eye disease has sufficient impact on daily life to justify the risks of immunosuppression (if active) or surgical intervention (if inactive).
  • Intravenous methylprednisolone in combination with oral mycophenolate sodium (or mofetil) represents the first-line treatment for moderate-to-severe and active GO.
  • In the more severe forms of moderate-to-severe and active GO, including constant/inconstant diplopia, severe inflammatory signs and exophthalmos > 25 mm, i.v. methylprednisolone at the highest cumulative dose (7.5 g per cycle) as monotherapy represents an additional valid first-line treatment.
  • Moderate-to-severe and active GO: hyperthyroidism should be treated with ATDs until treatment of GO is completed.
  • It is composed of entirely objective measures: ≥2-mm reduction of lid aperture, ≥1 point reduction in five-item CAS (excluding subjective, patient-reported spontaneous or gaze-evoked pain), ≥2 mm reduction in exophthalmos, ≥8° increase of eye muscle duction.
  • Improvement in ≥2 features in one eye without deterioration in the other eye might be considered a positive response to treatment.
  • Optimally, the outcome of treatment should be assessed 3 months after the last therapeutic intervention, but, in addition, changes after 6 months can also be considered.
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