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About 30% of UC patients eventually undergo colectomy for medically refractory disease [4].
In a randomized, placebo-controlled, phase III trial involving 351 patients with relapsed or refractory disease, the addition of lenalidomide to standard dexamethasone therapy nearly tripled overall response rates compared with dexamethasone alone (58% vs.
Vasavada and his colleagues are still accumulating data on Botoxuse in patients with overactive bladder, but the interim analysis shows that the mean number of weekly incontinence episodes dropped from 30 to 20 during a 3-month period following Botox injections in patients who have refractory disease.
All patients had relapsed or refractory disease and were heavily pretreated, with a median of four prior systemic therapies.
Forty-five % of patients had primary refractory disease and 55% progressed after responding to frontline therapy, among whom 90% received standard of care frontline treatment ABVD (Adriamycin, bleomycin, vinblastine and dacarbazine).
It is also a refractory disease progressing to AML for some patients with poor prognosis.
Leung shared insights from his clinical experience at National Jewish Health in Denver, whose pediatric eczema program represents a national referral center for patients with severely refractory disease and their families.
According to the company, the patients were required to have documented refractory disease, or relapsed disease.
The open label trial enrolled 266 patients who had relapsed or refractory disease and had received at least two prior lines of therapy, including a proteasome inhibitor and an IMiD--either thalidomide or lenalidomide (Revlimid).
In addition, the cohort treated with IVIG had a clinical improvement in mRSS that was similar to a group of patients treated with MMF who were typically deemed clinical responders, suggesting that addition of IVIG may stabilize previous refractory disease," they added.
The B-cell-depleting monoclonal antibody rituximab also has now been used in a small number of patients with refractory disease.
Despite the widespread availability and uptake of first-, second-, and third-generation antiepileptic drugs (AEDs), approximately one-third of patients with epilepsy suffer from refractory disease, leaving significant commercial opportunity for new and emerging drugs.

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