RBC Transfusions and Iron Chelation Therapy in Clinical Practice in MDS: A One Month Survey by the GFM.
Authors: Rose Christian , Stamatoulas A. , Vassilief D. , Brechignac S. , Beyne-Rauzy O. , Courtois F. , Wemeau M. , Guerci Agnes , Chaury M.P. , Lefs B. , Mahe B. , Roy L. , Quesnel Bruno , Dreyfus F. , Fenaux Pierre 
Published: October 2019

Background: RBC transfusions remain a mainstay in the treatment in MDS, but their characteristics in a large MDS population have not been reported in detail. Indications of iron chelation therapy (CT) in those patients, are based on consensus (Gattermann Hematology/Oncology Clinics 2005;19:supp1 ) but have not been reported in a large MDS cohort. In addition, a positive effect of desferioxamine (DFO) therapy on hematopoiesis has been reported in a small group of MDS (Jensen BJH, 1996;94:288).

Methods: We analyzed the hematological characteristics, RBC transfusion requirement and iron CT in MDS patients referred for RBC transfusion in 18 French centers between May 15 and June 15, 2005.

Results: 170 pts were included in the survey, median age 75 (14–95); M/F ratio 1.37; median time from diagnosis was 43 months (1 month to 17.5 years); WHO: pure RA 8.8%, pure RARS 18%, RCMD 3.5%, RCMD-RS 3%, RAEB I 16.5%, RAEB II 5.3%, 5q- syndrome 7%, CMML 4.7%, unclassified12.6%. Karyotype: fav (12%), int (37%), unfav ( 23%), failure or not done ( 28%). IPSS: low 39%, Int1 45%, Int2 13.5%, high 2.5%, unavailable 28%. Median serum ferritin (SF) level at diagnosis was 562ng/ml (9–2500). Median number of RBC units transfused since diagnosis was 49, mean 80 (2–610) and did not differ statistically according to WHO or IPSS. 81% of patients had received > 21 RBC units. Median number of RBC units transfused at initiation of CT was 21 (2–151). 68/170 pts ( 40 %) had received CT during the disease course including: DFO continuous s/c (8h) (40mg/kg/d, 3 to 5d/ week) n = 37, DFO s/c bolus (2 to 8g/week) n = 14, DFO IV (50 to 100mg/kg/d once after each RBC transfusion) n=7, deferiprone alone (30 to 75 mg/kg/d) n=4. Deferiprone + DFO s/c n=6. Median SF level was 1492ng/ml at the start of iron CT (436–6572). 50/68 pts were still on iron CT at the time of the survey after 2 to 114 months. Median SF level at the time of survey was 1828ng/ml (not statistically different from pre CT level). No positive effects were observed on hematopoiesis even in pts chelated for prolonged periods. 18/68 patients stopped CT (all were on DFO therapy) due to over efficacy n= 1, local complications n = 7, patient decision n=10 ). Median duration of treatment was 9 months (1–78) and median level of SF 3227ng/ml at the time of survey in those 18pts. For the 102/170 pts who did not receive CT, main reasons given by physicians were: older age 33% (median age in this group was indeed 82); cumbersome treatment 12%; high IPSS 8% (all cases had indeed IPSS>=1.5); low transfusion requirement 8% (median=11 RBC units transfused in this group); other reasons 5% (refusal, skin lesions), no reason given 34%. By comparison to non chelated patients, chelated patients had significantly higher number of RBC units transfused (mean 115 vs 55) (p<0.001), lower age (p= 0.002) but no difference in WHO or IPSS.

Conclusions: In transfused MDS pts, rarely analyzed, the number of RBC units previously transfused was not influenced by WHO or IPSS. This survey confirms that chelated patients are younger and more transfused. Despite heterogeneity in iron CT, efficacy based on the SF level seems to be good. No effect of iron CT on hematopoiesis was observed.