Table 1. Clinical and hematologic... | American Society of Hematology

Table 1.

Clinical and hematologic characteristics of patients with MDS/sAML treated with VPA ± ATRA

No	Study group	Sex	Age, y	WHO subtype¹³	MDS duration, mo	Karyotype	IPSS subgroup¹⁴	Prior treatment	Time since pretreatment, mo	Growth factor	Response	Response duration, mo	Time to response, d	Duration VPA, mo	Duration ATRA, mo
1	2	F	77	RAEB I	26	del5[16/23],+8[3/23]	Int II	Epo	2	No	SD	—	—	3	3
2	2	M	70	sAML/MDS	6	Complex	High	Thalidomide, gemcitabine	< 1	No	PD	—	—	4	4
3	1	M	72	RCMD	11	-Y[4/21]	Int I	Thalidomide	< 1	No	MaR-N*	5	38	8	—
4	2	M	57	RCMD	18	del9[15/20]	Int I	AraC, idarubicin	12	No	PD	—	—	3	3
5	1	F	64	RCMD with RS	9	del5[5/20],+8[12/20]	Int I	Thalidomide	2	No	PD	—	—	6	2
6	1	M	64	RAEB II	3	Complex	Int II	Thalidomide	< 1	No	PD	—	—	4	—
7	2	F	68	RCMD	8	Normal	Int I	No	—	No	PD	—	—	3	3
8	1	M	60	RCMD	100	Complex	Int I	Epo, thalidomide	1	No	SD	—	—	12	—
9	1	F	65	RCMD	23	Normal	Int I	2-CDA, Epo, thalidomide	2	No	SD	—	—	3	—
10	1	M	73	RCMD with RS	49	del11,-Y[19/22]	Int I	ATG, Epo/G-SCF	14	Epo	MiR-E	5	NA	10	3
11	1	M	67	RCMD	8	Normal	Int I	Thalidomide	1	No	PR	4	14	8	3
12	2	M	62	sAML/MDS	5	+8[4/21]	High	Thalidomide	< 1	No	SD†	—	—	3	9
13	1	M	67	RAEB II	22	Normal	Int II	Epo, thalidomide	< 1	Epo	MaR-P†	3	30	23‡	18‡
14	1	F	72	RCMD	52	Normal	Low	2 × ATG	6	No	MaR-E	9§	17	8‡	—
15	1	M	78	RCMD	70	Normal	Int I	Thalidomide, gemcitabine	< 1	No	PD	—	—	4	1
16	1	M	63	RCMD	24	-Y[5/20]	Low	ATG	22	No	MaR-P	5	35	22‡	15‡
17	1	F	72	sAML/MDS	25	Normal	High	Idarubicin/cytarabine, MTC	2	No	PD	—	—	3	1
18	1	M	67	RAEB I	28	Normal	Int I	No	—	No	SD	—	—	8‡	5‡
19	1	M	72	RAEB II	11	Normal	Int II	FTI	2	No	PD*	—	—	2	—
20	1	M	68	CMML	11	Normal	Int I	No	—	No	PD	—	—	4	2
21	1	M	74	sAML/MDS	12	92,XXYY[3/22]	High	No	—	No	MiR-E†	4§	27	6‡	4
22	1	M	72	RCMD	18	-20[5/22]	Int I	No	—	No	SD	—	—	3	1
23	1	M	35	PSA	122	Normal	Low	No	—	No	MaR-E	4§	31	5‡	—

No	Study group	Sex	Age, y	WHO subtype¹³	MDS duration, mo	Karyotype	IPSS subgroup¹⁴	Prior treatment	Time since pretreatment, mo	Growth factor	Response	Response duration, mo	Time to response, d	Duration VPA, mo	Duration ATRA, mo
1	2	F	77	RAEB I	26	del5[16/23],+8[3/23]	Int II	Epo	2	No	SD	—	—	3	3
2	2	M	70	sAML/MDS	6	Complex	High	Thalidomide, gemcitabine	< 1	No	PD	—	—	4	4
3	1	M	72	RCMD	11	-Y[4/21]	Int I	Thalidomide	< 1	No	MaR-N*	5	38	8	—
4	2	M	57	RCMD	18	del9[15/20]	Int I	AraC, idarubicin	12	No	PD	—	—	3	3
5	1	F	64	RCMD with RS	9	del5[5/20],+8[12/20]	Int I	Thalidomide	2	No	PD	—	—	6	2
6	1	M	64	RAEB II	3	Complex	Int II	Thalidomide	< 1	No	PD	—	—	4	—
7	2	F	68	RCMD	8	Normal	Int I	No	—	No	PD	—	—	3	3
8	1	M	60	RCMD	100	Complex	Int I	Epo, thalidomide	1	No	SD	—	—	12	—
9	1	F	65	RCMD	23	Normal	Int I	2-CDA, Epo, thalidomide	2	No	SD	—	—	3	—
10	1	M	73	RCMD with RS	49	del11,-Y[19/22]	Int I	ATG, Epo/G-SCF	14	Epo	MiR-E	5	NA	10	3
11	1	M	67	RCMD	8	Normal	Int I	Thalidomide	1	No	PR	4	14	8	3
12	2	M	62	sAML/MDS	5	+8[4/21]	High	Thalidomide	< 1	No	SD†	—	—	3	9
13	1	M	67	RAEB II	22	Normal	Int II	Epo, thalidomide	< 1	Epo	MaR-P†	3	30	23‡	18‡
14	1	F	72	RCMD	52	Normal	Low	2 × ATG	6	No	MaR-E	9§	17	8‡	—
15	1	M	78	RCMD	70	Normal	Int I	Thalidomide, gemcitabine	< 1	No	PD	—	—	4	1
16	1	M	63	RCMD	24	-Y[5/20]	Low	ATG	22	No	MaR-P	5	35	22‡	15‡
17	1	F	72	sAML/MDS	25	Normal	High	Idarubicin/cytarabine, MTC	2	No	PD	—	—	3	1
18	1	M	67	RAEB I	28	Normal	Int I	No	—	No	SD	—	—	8‡	5‡
19	1	M	72	RAEB II	11	Normal	Int II	FTI	2	No	PD*	—	—	2	—
20	1	M	68	CMML	11	Normal	Int I	No	—	No	PD	—	—	4	2
21	1	M	74	sAML/MDS	12	92,XXYY[3/22]	High	No	—	No	MiR-E†	4§	27	6‡	4
22	1	M	72	RCMD	18	-20[5/22]	Int I	No	—	No	SD	—	—	3	1
23	1	M	35	PSA	122	Normal	Low	No	—	No	MaR-E	4§	31	5‡	—

Study group 1 indicates VPA monotherapy; study group 2, VPA + ATRA; WHO, World Health Organization; IPSS, International Prognostic Scoring System; RAEB, refractory anemia with excess of blasts; Int, intermediate; Epo, erythropoietin; SD, stable disease; —, not applicable; PD, progressive disease; RCMD, refractory cytopenia with multilineage dysplasia; MaR-N, major neutrophil response; RCMD with RS, refractory cytopenia with multilineage dysplasia and ringed sideroblasts; 2-CDA, 2-chlorodeoxyadenosine; ATG, antithymocyte globulin; G-CSF, granulocyte colony-stimulating factor; MiR-E, minor erythroid response; NA, not applicable; PR, partial remission; MaR-P, major platelet response; MaR-E, major erythroid response; MTC, mitoxantrone, topotecan, cyclophosphamide; FTI, famesyltransferase inhibitor; CMML, chronic myelomonocytic leukemia; PSA, pure sideroblastic anemia.

Temporary rise in platelet count (< 2 months).

†

Reduction in blast count.

‡

Treatment ongoing.

Response ongoing.

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