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. 2019 Mar 7;133(10):1020-1030.
doi: 10.1182/blood-2018-06-857102. Epub 2018 Nov 7.

Proposals for revised IWG 2018 hematological response criteria in patients with MDS included in clinical trials

U Platzbecker  1   2 , P Fenaux  2   3 , L Adès  2   3 , A Giagounidis  2   4 , V Santini  2   5 , A A van de Loosdrecht  2   6 , D Bowen  7 , T de Witte  8 , G Garcia-Manero  9 , E Hellström-Lindberg  10 , U Germing  2   11 , R Stauder  12 , L Malcovati  13 , Mikkael A Sekeres  14 , David P Steensma  15 , S Gloaguen  2
Affiliations

Proposals for revised IWG 2018 hematological response criteria in patients with MDS included in clinical trials

U Platzbecker et al. Blood. .

Abstract

The heterogeneity of myelodysplastic syndromes (MDSs) has made evaluating patient response to treatment challenging. In 2006, the International Working Group (IWG) proposed a revision to previously published standardized response criteria (IWG 2000) for uniformly evaluating clinical responses in MDSs. These IWG 2006 criteria have been used prospectively in many clinical trials in MDSs, but proved challenging in several of them, especially for the evaluation of erythroid response. In this report, we provide rationale for modifications (IWG 2018) of these recommendations, mainly for "hematological improvement" criteria used for lower-risk MDSs, based on recent practical and reported experience in clinical trials. Most suggestions relate to erythroid response assessment, which are refined in an overall more stringent manner. Two major proposed changes are the differentiation between "procedures" and "criteria" for hematologic improvement-erythroid assessment and a new categorization of transfusion-burden subgroups.

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Conflict of interest statement

Conflict-of-interest disclosure: U.P. and P.F. have received renumeration and research funding from Celgene Corporation. The remaining authors declare no competing financial interests.

Figures

Figure 1.
Figure 1.
Transfusion history of 5 hypothetical MDS patients within 8 weeks. All receiving RBC support for an Hb of <9 g/dL for up to 8 weeks (56 days) prior to day 0, that is, start for a subsequent therapy, for example, within a clinical trial. The total number of RBC U within 8 weeks is 4 in all patients (Pat.).
Figure 2.
Figure 2.
Transfusion history of 5 hypothetical MDS patients within 16 weeks. Transfusion history of the same 5 hypothetical MDS patients (Figure 1) but with an extended period of 16 weeks (112 days) prior to the start of a putative therapy. The total number of RBC U within 8 weeks is <4 in patients 2, 3, and 4, respectively.
Figure 3.
Figure 3.
Hb measurements with 2 different devices or laboratories at different points in time in 1 patient treated, for example, with ESA. The figure illustrates that certain fluctuations of Hb values might be simply a result of differences in the accuracy of blood devices/methods. An Hb increase by ≥1.5 g/dL is achieved constantly only with device 1, which was also used during screening.
Figure 4.
Figure 4.
Undesired effects of dosing interruption. The figure shows the undesired effects of dosing interruption followed by dose reduction in a patient responding to ESA therapy and reaching an Hb ≥ 12 g/dL (starting from a baseline Hb of 8.5 g/dL). The lower line marks the IWG 2006 response (8.5-10 g/dL) and upper line the general Hb cutoff for dose interruption in many clinical trials (12 g/dL).
See this image and copyright information in PMC

References

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