Corticosteroid plus IV cyclophosphamide does not improve survival in acute exacerbation of IPF
Treatment with corticosteroids (CS) plus intravenous cyclophosphamide (IVCY) for a first episode of idiopathic acute exacerbation (AE) does not significantly improve short-term survival as compared with CS monotherapy in patients with idiopathic pulmonary fibrosis (IPF), a study has found.
“[T]he propensity score‐matched analysis demonstrated that for patients with IPF with a first idiopathic AE, first‐line therapy with CS + IVCY, compared with CS monotherapy, did not significantly improve post‐AE 90‐day survival rates,” the researchers said.
A total of 102 consecutive patients with AE-IPF were included. Post-AE survival rates and treatment safety were retrospectively examined. A propensity score-matched analysis was used to compare the efficacy of CS + IVCY therapy for a first AE episode with that of CS monotherapy.
The entire cohort had a 64.7-percent post-AE 90-day survival rate. Twenty-six matched patient pairs were formed based on propensity scores. Characteristics were well balanced between matched patients with AE-IPF treated with CS and those with CS + IVCY.
There were no significant between-group differences in post-AE 90-day survival rates (84.6 percent vs 76.9 percent; p=0.70), cumulative survival rates (p=0.57 by log-rank test) or incidence of adverse events ≥ CTCAE (Common Terminology Criteria for Adverse Events) v5.0 grade 3 (61.5 percent vs 65.4 percent; p=1.00). [Respirology 2019;24:792-798]
“Therefore, these results would not support the routine concurrent use of IVCY with CS as a first‐line therapy for AE‐IPF,” the researchers said. “We believe that our results could inform the design of future studies to establish optimal therapeutic strategies for patients with AE‐IPF.”
The efficacy of CS + IVCY therapy for AE-IPF was analysed in only two retrospective studies. The 90-day survival rates in patients who received CS + IVCY in these investigations were 55 percent and 73 percent, respectively, with few adverse events and safe profile. [Eur Respir J 2011;38:1487-1489; Sarcoidosis Vasc Diffuse Lung Dis 2016;33:385-391]
Although both studies suggested the potential benefit of CS + IVCY therapy, these were noncomparative by design and had small samples. On the other hand, the current study compared the efficacy of first-line therapy with CS + IVCY and that of CS monotherapy in a relatively larger AE-IPF cohort using a propensity score-matched analysis, “which is a strength of this study,” according to the researchers.
AE-IPF is a fatal event, with a 90-day survival rate ranging approximately from 20–70 percent. [Eur Respir J 2011;37:356-363; Respirology 2018;23:206-212; Lung 2014;192:141-149; Respir Res 2013;14:73]
“Although the reported short‐term survival rate is variable, it likely depends on differences in the diagnostic criteria of AE‐IPF, baseline characteristics of the study population, study design and supportive therapy, among others,” the researchers said.
The present study has several limitations, including its retrospective design, the inclusion of Japanese patients, “which necessitates further studies to confirm whether this result applies to other ethnic groups,” a sample size that might have been small to detect a significant between-group difference in survival, and potential underestimation of minor adverse events.