Abstract
In the CONVERT study, treatment with amikacin liposome inhalation suspension (ALIS) added to guideline-based therapy (GBT) met the primary end point of increased culture conversion by month 6 in patients with treatment-refractory Mycobacterium avium complex (MAC) lung disease (ALIS+GBT, 29% [65/224] vs GBT-alone, 8.9% [10/112], P<.0001). In patients who had culture conversion by month 6 in CONVERT, was conversion sustained (negative sputum cultures for 12 months on treatment) and durable (negative sputum culture for 3 months off treatment), and were there any additional safety signals associated with a full treatment course of 12 months post-conversion? Adults were randomized 2:1 to receive ALIS+GBT or GBT-alone. Patients achieving culture conversion by month 6 continued therapy for 12 months followed by off-treatment observation. More patients randomized to ALIS+GBT (intention-to-treat) had conversion that was both sustained and durable 3 months off treatment vs patients randomized to GBT-alone (ALIS+GBT, 16.1% [36/224] vs GBT-alone, 0% [0/112], P<.0001). Of the patients who had culture conversion by month 6, 55.4% of converters (36/65) in the ALIS+GBT-treated arm vs no converters (0/10) in the GBT-alone arm had sustained and durable conversion (P=.0017). Relapse rates through 3 months off treatment were 9.2% (6/65) in the ALIS+GBT and 30.0% (3/10) in the GBT-alone arm. Common adverse events among ALIS+GBT-treated patients (dysphonia, cough, dyspnea, hemoptysis) mainly occurred within the first 8 months of treatment. In a refractory population, conversion was sustained and durable in more patients treated with ALIS+GBT for 12 months postconversion than in those treated with GBT-alone. No new safety signals were associated with 12 months of postconversion treatment.
Highlights
Study Question: Are the microbiologic benefits of amikacin liposome inhalation suspension (ALIS) added to guideline-based therapy (GBT) in patients with refractory Mycobacterium avium complex (MAC) lung disease sustained over the 12-month postconversion treatment duration and are the treatment effects durable at 3 months after all MAC treatment? Results: Compared with patients treated with GBT alone, most ALIS-treated patients showed a sustained response at the completion of 12 months of postconversion treatment (63.1% ALIS plus GBT vs 30.0% GBT alone; P 1⁄4 .0644) and continued to show negative culture results 3 months after all MAC treatment (55.4% vs 0.0%; P 1⁄4 .0017)
In the treatment-refractory population of patients who participated in the CONVERT study,[16] nearly onethird achieved culture conversion by month 6 with ALIS added to GBT compared with < 10% of those treated with only GBT,[16] an encouraging outcome given the duration of participants’ illnesses and the lack of response to GBT before study entry
We further evaluated the efficacy of ALIS treatment by examining the sustainability of culture conversion through 12 months of treatment and the durability of conversion at 3 months after all MAC treatment
Summary
In the CONVERT study, treatment with amikacin liposome inhalation suspension (ALIS) added to guideline-based therapy (GBT) met the primary end point of increased culture conversion by month 6 in patients with treatment-refractory Mycobacterium avium complex lung disease (ALIS plus GBT, 29% [65/224] vs GBT alone, 8.9% [10/112]; P < .0001). RESULTS: More patients randomized to ALIS plus GBT (intention-to-treat population) achieved conversion that was both sustained and durable 3 months after treatment vs patients randomized to GBT alone (ALIS plus GBT, 16.1% [36/224] vs GBT alone, 0% [0/ 112]; P < .0001). Of the patients who achieved culture conversion by month 6, 55.4% of converters (36/65) in the ALIS plus GBT treated arm vs no converters (0/10) in the GBT alone arm achieved sustained and durable conversion (P 1⁄4 .0017). INTERPRETATION: In a refractory population, conversion was sustained and durable in more patients treated with ALIS plus GBT for 12 months after conversion than in those treated with GBT alone.
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