Table 3. Principal randomized clinical... | American Society of Hematology

Table 3.

Principal randomized clinical trials and observational studies evaluating occurrence of AML/MDS in PV patients

Study/year of publication	Strategies	N	Median follow-up, y	Cases of AL	Estimated incidence of AL, %	Estimated annual incidence rate
Randomized clinical trials
PVSG-01, 1981^1,19	Phlebotomy	134	8.6	2	1.5	0.17
	Chlorambucil	141		19	13	1.5
	P32	156		15	9.6	1.1
EORTC, 1981²	P32	140	8	2	1.4	0.17
	Busulphan	145		3	2.0	0.25
FPSG, 1997 (older than 65)⁴	P32	242	16, maximum	35	12 at 10 y*	1.2
	P32 + HU	219			21 at 10 y*	2.1
FSPG, 1997 (older than 65)⁵	HU	150	16, maximum	13	5 at 10 y*	0.5
	Pipobroman	142			Similar in both groups
Observational prospective studies
PVSG-08, 1986^6,7	HU	51	8.6	5	9.8	1.1
Weinfeld et al, 1994⁸	HU	30	10	3	10	1
Tarstarsky and Sharon, 1997⁹	HU	71	10.9	4	5.6	0.5
Brusamolino et al, 1984¹⁰	Pipobroman	100	5	6	6	1.2
Passamonti et al, 2000¹¹	Pipobroman	163	10	11	7	0.7
Kiladjian et al, 2003¹²	Pipobroman	164	11.4	32	19.5	1.7
Passamonti et al, 2003 (younger than 50)²⁴	Pipobroman, 73% HU, 9% Phlebotomy, 18%	70	14	5	7	0.5
ECLAP, 2004	Phlebotomy, 63% HU, 48% P32, 3% Interferon, 4% Alkylators, 10%	1638	2.8	22	1.3	0.5
Observational retrospective studies
Halnan and Russell, 1965²⁸	No P32	127	19, maximum	0	0	0
	P32	107		0
Najean et al, 1988¹⁴	Phlebotomy, 22% P32, 61% Cytoreductive drugs, 17%	288	10, minimum	33	11.4	1.1
GISP, 1995²⁵	Phlebotomy, 31% Alkylators, 59% Other cytoreductive drugs, 10%	1213	5.3	28	2.3	0.43
Brodman et al, 2000²⁶	HU, 63% Alkylators, 19% P32, 5% Phlebotomy, 70%	59	3.7	1	1.7	0.5
Nielsen and Hasselbalch, 2003²⁷	No HU	13	8	1	7.7	0.9
	HU, alone or with busulphan	29		7	24	3.0

Study/year of publication	Strategies	N	Median follow-up, y	Cases of AL	Estimated incidence of AL, %	Estimated annual incidence rate
Randomized clinical trials
PVSG-01, 1981^1,19	Phlebotomy	134	8.6	2	1.5	0.17
	Chlorambucil	141		19	13	1.5
	P32	156		15	9.6	1.1
EORTC, 1981²	P32	140	8	2	1.4	0.17
	Busulphan	145		3	2.0	0.25
FPSG, 1997 (older than 65)⁴	P32	242	16, maximum	35	12 at 10 y*	1.2
	P32 + HU	219			21 at 10 y*	2.1
FSPG, 1997 (older than 65)⁵	HU	150	16, maximum	13	5 at 10 y*	0.5
	Pipobroman	142			Similar in both groups
Observational prospective studies
PVSG-08, 1986^6,7	HU	51	8.6	5	9.8	1.1
Weinfeld et al, 1994⁸	HU	30	10	3	10	1
Tarstarsky and Sharon, 1997⁹	HU	71	10.9	4	5.6	0.5
Brusamolino et al, 1984¹⁰	Pipobroman	100	5	6	6	1.2
Passamonti et al, 2000¹¹	Pipobroman	163	10	11	7	0.7
Kiladjian et al, 2003¹²	Pipobroman	164	11.4	32	19.5	1.7
Passamonti et al, 2003 (younger than 50)²⁴	Pipobroman, 73% HU, 9% Phlebotomy, 18%	70	14	5	7	0.5
ECLAP, 2004	Phlebotomy, 63% HU, 48% P32, 3% Interferon, 4% Alkylators, 10%	1638	2.8	22	1.3	0.5
Observational retrospective studies
Halnan and Russell, 1965²⁸	No P32	127	19, maximum	0	0	0
	P32	107		0
Najean et al, 1988¹⁴	Phlebotomy, 22% P32, 61% Cytoreductive drugs, 17%	288	10, minimum	33	11.4	1.1
GISP, 1995²⁵	Phlebotomy, 31% Alkylators, 59% Other cytoreductive drugs, 10%	1213	5.3	28	2.3	0.43
Brodman et al, 2000²⁶	HU, 63% Alkylators, 19% P32, 5% Phlebotomy, 70%	59	3.7	1	1.7	0.5
Nielsen and Hasselbalch, 2003²⁷	No HU	13	8	1	7.7	0.9
	HU, alone or with busulphan	29		7	24	3.0

Estimated annual incidence rate is based on number of cases per 100 persons per year.

Actuarial risk.