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2018 ; 2
(14
): 1765-1772
Nephropedia Template TP
gab.com Text
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English Wikipedia
The use of immunosuppressive therapy in MDS: clinical outcomes and their
predictors in a large international patient cohort
#MMPMID30037803
Stahl M
; DeVeaux M
; de Witte T
; Neukirchen J
; Sekeres MA
; Brunner AM
; Roboz GJ
; Steensma DP
; Bhatt VR
; Platzbecker U
; Cluzeau T
; Prata PH
; Itzykson R
; Fenaux P
; Fathi AT
; Smith A
; Germing U
; Ritchie EK
; Verma V
; Nazha A
; Maciejewski JP
; Podoltsev NA
; Prebet T
; Santini V
; Gore SD
; Komrokji RS
; Zeidan AM
Blood Adv
2018[Jul]; 2
(14
): 1765-1772
PMID30037803
show ga
Most studies of immunosuppressive therapy (IST) in myelodysplastic syndromes
(MDS) are limited by small numbers and their single-center nature, and report
conflicting data regarding predictors for response to IST. We examined outcomes
associated with IST and predictors of benefit in a large international cohort of
patients with MDS. Data were collected from 15 centers in the United States and
Europe. Responses, including red blood cell (RBC) transfusion independence (TI),
were assessed based on the 2006 MDS International Working Group criteria, and
overall survival (OS) was estimated by Kaplan-Meier methods. Logistic regression
models estimated odds for response and TI, and Cox Proportional Hazard models
estimated hazards ratios for OS. We identified 207 patients with MDS receiving
IST, excluding steroid monotherapy. The most common IST regimen was
anti-thymocyte globulin (ATG) plus prednisone (43%). Overall response rate (ORR)
was 48.8%, including 11.2% (95% confidence interval [CI], 6.5%-18.4%) who
achieved a complete remission and 30% (95% CI, 22.3%-39.5%) who achieved RBC TI.
Median OS was 47.4 months (95% CI, 37-72.3 months) and was longer for patients
who achieved a response or TI. Achievement of RBC TI was associated with a
hypocellular bone marrow (cellularity < 20%); horse ATG plus cyclosporine was
more effective than rabbit ATG or ATG without cyclosporine. Age, transfusion
dependence, presence of paroxysmal nocturnal hemoglobinuria or large granular
lymphocyte clones, and HLA DR15 positivity did not predict response to IST. IST
leads to objective responses in nearly half the selected patients with the
highest rate of RBC TI achieved in patients with hypocellular bone marrows.