| Studies examining iron overload and organ endpoints in transfusion dependent lower IPSS risk MDS patients. | |||||
|---|---|---|---|---|---|
| Study | N | IOL measure | Endpoint | Findings | Reference |
| Schaefer 1981 | 15 | Median RBCU 120 | organs | Elevated LFTs (87%), portal fibrosis (66%), cardiomegaly (50%), CHF (27%), arrhythmias (53%), abnormal GTT (100%) | [1] |
| Jaeger 1992 | 46 | Median RBCU 79 | organs | Elevated LFTs (24%), cardiac siderosis (43%), CHF deaths (30%), arrhythmias (22%), DM (11%) | [2] |
| Cazzola 1988 | 26 | Median RBCU 64 | organs | Elevated LFTs (69%), CHF (31%), impaired GTT/DM (77%) | [3] |
| Takatoku 2007 | 152 | Median RBCU ≈ 100 | organs | Elevated LFTs (90%), hepatic failure (7%)*, CHF (24%)* | [4] |
| Ferte 2006 | 21 | Median RBCU 81 | heart | Cardiomegaly (43%), CHF (66%) | [5] |
| *present at death. CHF, congestive heart failure; DM, diabetes mellitus; GTT, glucose tolerance test; IOL, iron overload; IPSS, International Prognostic Scoring System; LFT, liver function tests (transaminases); n, number; RBCU, red blood cell units; Modified from Wells RA, et al. Leuk Res. 2008;32:1338-53 and Leitch HA, et al. Leuk Res. 2018;74:21-41. | |||||
| Studies examining organ endpoints in transfusion dependent patients with lower IPSS risk MDS by receipt of iron chelation therapy. | |||||||
|---|---|---|---|---|---|---|---|
| Study | N | Design | Endpoint | Non-chelated patients | Chelated patients | p value | Reference |
| Jensen 20031 | 29 | Observational | Organ - hepatic | NA | Baseline ↑ ALT in 16 Normalized at 1y in 13 | NR | [1] |
| Jensen 19971 | 11 | Observational | Organ - cardiac | NA |
Baseline ↑ cardiac iron in 8 Normalized at 6-10 mo in 10 |
NR | [2] |
| Rose 2010 | 97 | Prospective follow-up | Cardiac mortality | 34.5% | 37% | NS | [3] |
| Remacha 2012, 2015 | 263 | Retrospective | Cardiac EFS | 90 mo | 137 mo | 0.017 | [4,5] |
| Lyons 20172 | 599 | Prospective, registry | New or progressive cardiac condition | NR | NR | NS | [6] |
| Gattermann 2012 | 71 | Prospective, pooled data |
Organ – hepatic LIC decrease >30% Normal ALT |
NA |
With baseline LIC ≥7 mg/gDW: 58.9% 71.4% at EOS vs 32.1% baseline |
NR | [7] |
| Wong 20183 | 151 | Retrospective |
Time to cardiac event MVA |
7 mo |
20 mo HR for shorter TTCE 0.93 [95% CI 0.87-0.99] |
0.02 0.03 |
[8] info |
| ALT, alanine aminotransferase; ARR, arrythmia; CAD, coronary artery disease; CHF, congestive heart failure; CM, comorbidities; CVS, cardiovascular; DFO, deferoxamine; DW, dry weight; EFS, event-free survival; EOS, end of study; HR, hazard ratio; IPSS, International Prognostic Scoring System; LIC, liver iron concentration; mo, months; MVA, multivariate analysis; N, number; NA, not applicable; NR, not reported; NS, not significant; TTCE, time to cardiac event; RBC, red blood cell; y, years. 1DFO. 2CHF or ARR. Similar results were seen in patients with & without concomitant CVS or endocrine CM. 3CHF, CAD or ARR. TTCE measured from first RBC transfusion dependence. Modified from Wells RA, et al. Leuk Res. 2008;32:1338-53 and Leitch HA, et al. Leuk Res. 2018;74:21-41. | |||||||