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Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
Dynamic assessment of RBC-transfusion dependency improves the prognostic value of the revised-IPSS in MDS patients
Devendra K Hiwase1,2,3,4, Deepak Singhal1,2,3, Corinna Strupp5
1Haematology Department, SA Pathology, Adelaide, Australia.
Abstract:
RBC-transfusion dependency (RBC-TD) is an independent prognostic factor for poor overall survival (OS) in the WHO classification-based prognostic scoring system (WPSS) for MDS patients. However, WPSS did not include cytopenia, whereas revised International Prognostic Scoring System (IPSS-R) did not include RBC-TD. Thus, neither of these prognostic scoring systems incorporates both cytopenia and RBC-TD. We aimed to test whether RBC-TD adds prognostic value to the IPSS-R. We analyzed MDS patients not treated with disease-modifying therapy, and enrolled in SA-MDS Registry (derivation cohort; n = 295) and Dusseldorf registry (Germany; validation cohort; n = 113) using time-dependent Cox proportional regression and serial landmark analyses. In the derivation cohort, RBC-TD patients had inferior OS compared to RBC transfusion-independent (RBC-TI) patients (P < 0.0001) at 6- (18 vs. 64 months), 12- (24 vs. 71 months), and 24-months (40 vs. 87 months). In a Cox proportional regression analysis, RBC-TD was an independent adverse prognostic marker in addition to age, sex, and IPSS-R variables (P < 0.0001). A prognostic index (PI) was derived using these Cox-proportional regression model variables. In the validation cohort, this PI classified patients into four prognostic groups with significantly different OS (P < 0.001) as in the derivation cohort. In conclusion, multivariate analysis by Cox proportional hazards regression and serial landmark analyses clearly demonstrates that development of RBC-TD at any time during the course of MDS is associated with poor OS, independent of IPSS-R. This study demonstrates that dynamic assessment of RBC-TD provides additional prognostic value to IPSS-R and should be included in treatment decision algorithms for MDS patients.
Insights
Red blood cell-transfusion dependency (RBC-TD) significantly worsens overall survival in myelodysplastic syndromes (MDS) patients. This study confirms RBC-TD is an independent prognostic factor, adding value to the IPSS-R scoring system for MDS patient management.
Area of Science:
- Hematology
- Oncology
- Clinical Prognostics
Background:
- Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders.
- Current prognostic scoring systems like the WHO classification-based prognostic scoring system (WPSS) and the revised International Prognostic Scoring System (IPSS-R) have limitations.
- WPSS does not include cytopenia, while IPSS-R omits red blood cell-transfusion dependency (RBC-TD).
Purpose of the Study:
- To evaluate whether RBC-TD provides additional prognostic value to the IPSS-R in MDS patients.
- To determine if RBC-TD is an independent prognostic marker for overall survival (OS) in MDS.
- To assess the utility of dynamic RBC-TD assessment in MDS treatment decision algorithms.
Main Methods:
- Analysis of MDS patients not treated with disease-modifying therapy from the SA-MDS Registry (derivation cohort, n=295) and Dusseldorf registry (validation cohort, n=113).
- Utilized time-dependent Cox proportional regression and serial landmark analyses.
- Developed and validated a prognostic index (PI) incorporating RBC-TD and IPSS-R variables.
Main Results:
- RBC-TD patients exhibited significantly inferior OS compared to RBC transfusion-independent (RBC-TI) patients in the derivation cohort.
- RBC-TD was identified as an independent adverse prognostic marker, alongside age, sex, and IPSS-R variables (P<0.0001).
- The derived PI effectively classified patients into four prognostic groups with significantly different OS in both derivation and validation cohorts.
Conclusions:
- The development of RBC-TD at any point during MDS is strongly associated with poor OS, independent of the IPSS-R.
- Dynamic assessment of RBC-TD offers significant additional prognostic value to the IPSS-R.
- Incorporating RBC-TD into treatment decision algorithms for MDS patients is recommended.

