Table 1.
Summary of retrospective studies demonstrating a statistically significant overall survival advantage in association with iron chelation therapy in patients with myelodysplastic syndromes and secondary iron overload in the non-stem cell transplantation setting.
| Study | Type of study | Participants (n) | Population | ICT agent | Median OS | Leukemic transformation | Infection-related deaths | Cardiac deaths | Hepatic deaths | Ref. |
|---|---|---|---|---|---|---|---|---|---|---|
| Leitch et al. | Retrospective | 178 (18 on ICT) | LR-MDS (99); HR-MDS (34) | DFO (18) | 40 months for non-ICT vs 226 months for ICT (p = 0.003) | 4 in non-ICT vs 1 in ICT (p = 0.06) | 2 deaths in non-ICT vs 1 death in ICT (p = 0.05) | 0 deaths in non-ICT vs 2 deaths in ICT (p = NS) | NS | [74] |
| Raptis et al. | Retrospective | 102 (41 on ICT) | LR-MDS (78, 32 on ICT); HR-MDS (24, 9 on ICT) | DFO (12); DFX (29) | 8.7 years for ICT vs 4.7 years for non-ICT (p = 0.03) | 4 in non-ICT vs 3 in ICT (p = NS) | NS | NS | NS | [6] |
| Rose et al. | Retrospective/prospective cohort | 97 (53 on ICT) | LR-MDS | DFO (40); DFX (4); DFP (5); DFP + DFO (4) | 53 months for non-ICT vs 124 months for ICT (p < 0.0003) | 15 in non-ICT vs 9 in ICT (p = 0.087) | 6 deaths in non-ICT vs 7 deaths in ICT (p = NS) | 10 deaths in non-ICT vs 10 deaths in ICT (p = NS) | I death in non-ICT vs 0 deaths in ICT | [5] |
| Neukirchen et al. | Retrospective matched-pair analysis | 188 (94 on ICT) | LR-MDS (78); HR-MDS (16) | DFO (53); DFX (47); DFO then DFX (14); DFO then DFP (3) | 49 months for non-ICT vs 75 months for ICT (p = 0.002) | 13 in non-ICT vs 12 in ICT (p = 0.73) | 12 deaths in non-ICT vs 33 deaths in ICT (p = 0.03) | NS | NS | [75] |
| Leitch et al. | Retrospective | 182 (38 on ICT) | LR-MDS (175) | DFO (9); DFX (19); DFO then DFX (9); DFX then DFO (1) | Non-RARS: 44 months for non-ICT vs NR for ICT (p < 0.0001). RARS: 99 months for non-ICT vs 134.4 months for ICT (p = 0.025). RA/del-5q: 60 months for non-ICT vs NR for ICT (p = 0.017) | Non-RARS: 10 leukemic deaths in non-ICT vs 0 leukemic deaths in ICT (p = NS). RARS: 2 leukemic deaths in non-ICT vs 1 leukemic death in ICT (p = NS). RA/ del-5q: 2 leukemic deaths in non-ICT vs 0 leukemic deaths in ICT (p = NS) | Non-RARS: 10 deaths in non-ICT vs 0 death in ICT (p = NS). RARS: 3 deaths in non-ICT vs 1 death in ICT (p = NS). RA/ del-5q: 2 deaths in non-ICT vs 0 deaths in ICT (p = NS) | Non-RARS: 1 death in non-ICT vs 2 deaths in ICT (p = NS). RARS: 0 deaths in non-ICT vs 2 deaths in ICT (p = NS). RA/ del-5q: 1 death in non-ICT vs 2 deaths in ICT (p = NS) | Non-RARS: 1 death in non-ICT vs 0 deaths in ICT (p = NS). RARS: no deaths in either group. RA/del-5q: no deaths in either group | [76] |
DFO: Deferoxamine; DFP: Deferipone; DFX: Deferasirox; HR-MDS: Higher-risk myelodysplastic syndromes; ICT: Iron chelation therapy; LR-MDS: Lower-risk myelodysplastic syndromes; NS: Not stated; NR: Not reached; OS: Overall survival; RARS: Refractory anemia with ringed sideroblasts; RA/del-5q: Refractory anemia/deletion of long arm of chromosome 5.