Evolution to secondary MDS/AML in PNH
| Study/Year . | Patients, n . | Median age (range), y . | Country, study period, study design . | Treatment regimen . | Median follow-up (range), y . | No. of patients transformed to MDS/AML and, if reported, the median time to transformation . | Projected incidence of transformation to MDS/AML . |
|---|---|---|---|---|---|---|---|
| de Latour et al20 /2008 | 454* (Classic PNH, n = 113; AA-PNH syndrome, n = 224; Int PNH, n = 93) | 34 (24-48) | France, 1950-2005, retrospective | Steroids, IST, BMT (n = 52) | 6.8 (SE 0.5) | Total (MDS or AML), n = 26 (5.7%); | 10-y rates: Overall, 3.8% (95% CI, 0.1% to 7.5%); |
| MDS: n = 21, 1.3 (IQR 0.5-4.8) y; | Classic PNH, 3.8% (95% CI, 0.1% to 7.5%); | ||||||
| Socié et al122 /1996 | AML: n = 9 (4 with prior MDS), 1.6 (IQR 0.8-4.4) y | AA-PNH, 9% (95% CI, 4.5% to 13.6%); | |||||
| Indeterminate PNH, 3.7% (95% CI, 0.1% to 7.5%) | |||||||
| Nishimura et al19 /2004 | 176 (US); | 30 (4-80); | USA/Japan, 1966-2004, retrospective | Transfusions, androgens, prednisone, ATG/ALG, CsA, EPO, G-CSF, BMT (n = 8) | 7.6 (NR); | MDS: n = 6 (3.4%), AL: n = 1 (0.6%); | NR |
| 209 (Japan) | 45 (10-86) | 8.5 (NR) | MDS: n = 8 (3.8%), AL: n = 6 (2.9%) | ||||
| Ghosh125 /2015 | 33 | NR | India, 2010s (3-y period), prospective | NR | NR | AL, n = 1 (prior MDS) (3%); 0.5 y | NR |
| Ware et al123 /1991 | 26 | 14 (0.8-21) | USA, 1966-1991, retrospective | Prednisone, androgens, ATG | 8.5† (2.8-27) | AL, n = 1 (3.8%); 6 y | NR |
| Harris et al124 /1999 | 1760‡ | NR | Multiple, 1962-1999, retrospective | NR | NR | Total (MDS or AML), n = 38 (2.2%); | NR |
| MDS, n = 25 (1.4%); | |||||||
| AL, n = 16 (3 with prior MDS) (0.9%) |
| Study/Year . | Patients, n . | Median age (range), y . | Country, study period, study design . | Treatment regimen . | Median follow-up (range), y . | No. of patients transformed to MDS/AML and, if reported, the median time to transformation . | Projected incidence of transformation to MDS/AML . |
|---|---|---|---|---|---|---|---|
| de Latour et al20 /2008 | 454* (Classic PNH, n = 113; AA-PNH syndrome, n = 224; Int PNH, n = 93) | 34 (24-48) | France, 1950-2005, retrospective | Steroids, IST, BMT (n = 52) | 6.8 (SE 0.5) | Total (MDS or AML), n = 26 (5.7%); | 10-y rates: Overall, 3.8% (95% CI, 0.1% to 7.5%); |
| MDS: n = 21, 1.3 (IQR 0.5-4.8) y; | Classic PNH, 3.8% (95% CI, 0.1% to 7.5%); | ||||||
| Socié et al122 /1996 | AML: n = 9 (4 with prior MDS), 1.6 (IQR 0.8-4.4) y | AA-PNH, 9% (95% CI, 4.5% to 13.6%); | |||||
| Indeterminate PNH, 3.7% (95% CI, 0.1% to 7.5%) | |||||||
| Nishimura et al19 /2004 | 176 (US); | 30 (4-80); | USA/Japan, 1966-2004, retrospective | Transfusions, androgens, prednisone, ATG/ALG, CsA, EPO, G-CSF, BMT (n = 8) | 7.6 (NR); | MDS: n = 6 (3.4%), AL: n = 1 (0.6%); | NR |
| 209 (Japan) | 45 (10-86) | 8.5 (NR) | MDS: n = 8 (3.8%), AL: n = 6 (2.9%) | ||||
| Ghosh125 /2015 | 33 | NR | India, 2010s (3-y period), prospective | NR | NR | AL, n = 1 (prior MDS) (3%); 0.5 y | NR |
| Ware et al123 /1991 | 26 | 14 (0.8-21) | USA, 1966-1991, retrospective | Prednisone, androgens, ATG | 8.5† (2.8-27) | AL, n = 1 (3.8%); 6 y | NR |
| Harris et al124 /1999 | 1760‡ | NR | Multiple, 1962-1999, retrospective | NR | NR | Total (MDS or AML), n = 38 (2.2%); | NR |
| MDS, n = 25 (1.4%); | |||||||
| AL, n = 16 (3 with prior MDS) (0.9%) |
Rates of transformation to MDS/AML in series of patients with PNH.
BMT, bone marrow transplantation; EPO, erythropoietin; Int PNH, intermediate PNH; SE, standard error. See Table 1 for expansion of other abbreviations.
Patients with documented follow-up.
Mean value.
Compiled 15 series of ≥20 PNH patients from 1965 through 1995; see references within.