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Clinical Trial
. 2012 Jan;47(1):125-31.
doi: 10.1038/bmt.2011.41. Epub 2011 Mar 7.

Prognostic value of response after upfront therapy for acute GVHD

R M Saliba  1 , D R Couriel, S Giralt, G Rondon, G-J Okoroji, A Rashid, R E Champlin, A M Alousi
Affiliations
Clinical Trial

Prognostic value of response after upfront therapy for acute GVHD

R M Saliba et al. Bone Marrow Transplant. 2012 Jan.

Abstract

One challenge in designing clinical trials for treatment of acute GVHD (aGVHD) is the lack of an established standardized end point to measure the success of therapies. To facilitate assessment of end points in clinical trials for treatment of aGVHD in the current allo-SCT era, a national workshop was recently organized. In this study, which was presented at the workshop, we evaluated the prognostic value of response to upfront therapy in a cohort of 83 patients who had been enrolled on two clinical trials testing novel therapies for aGVHD at our institution. Our results indicate that patients whose aGVHD has a CR or PR by day 28 after initiation of systemic therapy have a significantly lower 6-month cumulative incidence of non-relapse mortality (NRM) (16%) than patients whose aGVHD did not respond to therapy by day 28 (48%, P=0.005). Multivariate analysis based on the Cox proportional hazards regression analysis showed that the impact of response on NRM is independent of patient and aGVHD characteristics. Our data confirm the validity of using day-28 response as a primary end point in clinical trials for upfront therapy for aGVHD.

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Conflict of interest statement

Conflict of interest

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Distribution (%) of response to therapy for aGVHD over time. Patients who received salvage therapy were coded as ‘‘PD’’ irrespective of response to salvage. Response on day 28 was unknown in two patients who died at 3 and 15 months from study entry due to underlying malignancy and stroke, respectively. NE, non-evaluable: two patients died of aGVHD on days 12 and 15 from study entry and were not evaluable for day-28 assessment. MR, mixed response; NR, no response; PD, progressive disease.
Figure 2
Figure 2
Cumulative incidence of NRM according to aGVHD grade and response to upfront therapy.
See this image and copyright information in PMC

References

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