What is the expected duration of clinical benefit?

From the Guidelines

The expected duration of clinical benefit for autoimmune hepatitis patients on conventional corticosteroid therapy is approximately 22 months, with 77% of patients achieving clinical, laboratory, and histological resolution within 24 months. This timeframe can vary significantly among individuals, with some patients responding within 6 months and others requiring continuous therapy for more than 3 years 1. The decision to continue or modify treatment should be based on the patient's response to therapy, with consideration given to their age, serological status, and degree of residual disease activity. For example, young adults with autoimmune hepatitis may require longer treatment durations, while elderly patients may achieve remission more quickly 1.

Factors Influencing Treatment Duration

• Age: Young adults (<40 years) may require longer treatment durations, with only 64% achieving remission within 24 months, compared to 94% of patients aged ≥60 years 1.
• Serological status: The presence of certain serological markers, such as anti-SLA, may indicate a higher risk of treatment dependence and relapse 1.
• Degree of residual disease activity: Patients with ongoing disease activity may require longer treatment durations or alternative therapies, such as azathioprine 1.

Treatment Strategies

• Conventional corticosteroid therapy: The average duration of treatment is 22 months, with a range of 1-180 months 1.
• Alternative therapies: Azathioprine may be considered for patients who require long-term maintenance therapy or who have not responded to conventional corticosteroid therapy 1.
• Treatment withdrawal: Patients who have achieved clinical, laboratory, and histological remission may be considered for treatment withdrawal, regardless of age or serological status 1.

It is essential to note that individual responses to treatment can vary significantly, and the expected duration of clinical benefit may be influenced by a range of factors, including the patient's underlying condition, treatment regimen, and overall health status 1.

From the FDA Drug Label

The sustained efficacy of amlodipine in angina patients has been demonstrated over long-term dosing. The mean duration of follow-up was 19 months. In a long-term (follow-up at least 6 months, mean 13.8 months) placebo-controlled mortality/morbidity study of amlodipine 5 to 10 mg in 1153 patients with NYHA Classes III (n=931) or IV (n=222) heart failure on stable doses of diuretics, digoxin, and ACE inhibitors, amlodipine had no effect on the primary endpoint of the study which was the combined endpoint of all-cause mortality and cardiac morbidity Another study (PRAISE-2) randomized patients with NYHA Class III (80%) or IV (20%) heart failure without clinical symptoms or objective evidence of underlying ischemic disease, on stable doses of ACE inhibitors (99%), digitalis (99%), and diuretics (99%), to placebo (n=827) or amlodipine (n=827) and followed them for a mean of 33 months

The expected duration of clinical benefit of amlodipine is at least 6 months to 33 months as demonstrated in long-term studies 2.

From the Research

Expected Duration of Clinical Benefit

The expected duration of clinical benefit is a crucial aspect of treatment evaluation. However, there is limited direct evidence on this topic.

• The study 3 found that clinicians consistently overestimated the chance that treatments would benefit an individual patient, but it did not provide information on the expected duration of clinical benefit.
• The study 4 emphasized the importance of evaluating treatment benefits based on clinical outcomes, but it did not discuss the duration of clinical benefit.
• The study 5 compared the effects of two blood pressure-lowering drugs on cardiovascular events and death, but it did not provide information on the expected duration of clinical benefit.
• The study 6 found that clinicians rarely had accurate expectations of benefits or harms, with inaccuracies in both directions, but it did not discuss the duration of clinical benefit.
• The study 7 focused on clinician-reported outcome assessments of treatment benefit, but it did not provide information on the expected duration of clinical benefit.

Factors Influencing Clinical Benefit

Several factors can influence the clinical benefit of a treatment, including:

• The type of treatment or intervention used 5
• The patient's individual characteristics and health status 3, 6
• The clinician's expectations and perceptions of the treatment benefits and harms 3, 6
• The measurement properties of the outcome assessments used to evaluate treatment effects 7

Measurement of Clinical Benefit

The measurement of clinical benefit is critical to evaluating the effectiveness of treatments.

• The study 7 emphasized the importance of applying good measurement practices to clinician-reported outcome assessments, including defining the context of use, identifying the concept of interest measured, and evaluating measurement properties.
• The study 4 highlighted the need to focus on clinical outcomes that are relevant to patients when evaluating treatment benefits.