Characteristics of 130 eligible patients by treatment group
| Variables . | Cy . | Cy + ATG . | P* . | ||
|---|---|---|---|---|---|
| N eval . | N (%) . | N eval . | N (%) . | ||
| No. of patients | 60 | 70 | |||
| No. of centers | 27 | 29 | |||
| Median age, y (range) | 60 | 26 (4-51) | 70 | 23 (1-51) | .11 |
| Age at transplantation | 60 | 70 | .70 | ||
| Younger than 10 y | 5 (8) | 9 (13) | |||
| 10-19 y | 13 (22) | 20 (28) | |||
| 20-29 y | 18 (30) | 20 (28) | |||
| 30-39 y | 13 (22) | 12 (18) | |||
| At least 40 y | 11 (18) | 9 (13) | |||
| Male sex | 60 | 35 (58) | 70 | 46 (66) | .39 |
| Karnofsky score at least 90% | 60 | 32 (53) | 67 | 41 (61) | .37 |
| Prior treatment | 60 | 69 | .99 | ||
| None | 30 (50) | 37 (53) | |||
| ATG + CsA ± other | 4 (7) | 6 (8) | |||
| ATG ± other (not CsA) | 1 (1) | 1 (2) | |||
| CsA ± other (not ATG) | 9 (15) | 11 (16) | |||
| Corticosteroids | 4 (7) | 4 (6) | |||
| Corticosteroids + other | 5 (8) | 5 (7) | |||
| Other† | 7 (12) | 6 (8) | |||
| No. of donor exposures prior to transplantation | 60 | 70 | .04 | ||
| 0 | 3 (5) | 1 (2) | |||
| 1-10 | 20 (33) | 21 (30) | |||
| More than 10 | 30 (50) | 26 (37) | |||
| Transfusions given, unknown no. | 7 (12) | 22 (31) | |||
| Median nucleated cell dose, × 108/kg (range) | 44 | 2.4 (.01-5) | 59 | 2.7 (.01-7) | .82 |
| Donor-recipient sex match | 60 | 69 | .37 | ||
| Male → Male | 21 (35) | 26 (38) | |||
| Male → Female | 15 (25) | 9 (13) | |||
| Female → Male | 14 (23) | 20 (29) | |||
| Female → Female | 10 (17) | 14 (20) | |||
| Donor-recipient CMV status | 53 | 61 | .90 | ||
| Donor +/recipient + | 28 (53) | 29 (47) | |||
| Donor +/recipient − | 10 (19) | 11 (18) | |||
| Donor −/recipient + | 5 (9) | 6 (10) | |||
| Donor −/recipient − | 10 (19) | 15 (25) | |||
| Graft type | 60 | 69 | .56 | ||
| Bone marrow | 58 (96) | 68 (99) | |||
| PBS‡C | 1 (2) | 1 (1) | |||
| BM + PBSC‡ | 1 (2) | 0 | |||
| Median time from Dx to Tx, mo (range) | 60 | 2 (<1-109) | 69 | 2 (<1-70) | .63 |
| Time from Dx to Tx | 60 | 69 | .78 | ||
| Less than 3 mo | 41 (69) | 45 (65) | |||
| 3 to less than 6 mo | 9 (15) | 8 (12) | |||
| 6-12 mo | 5 (8) | 9 (13) | |||
| At least 12 mo | 5 (8) | 7 (10) | |||
| Year of transplantation | 60 | 69 | .65 | ||
| 1994-1995 | 16 (27) | 18 (26) | |||
| 1996-1997 | 23 (38) | 22 (32) | |||
| 1998-1999 | 13 (22) | 14 (20) | |||
| 2000-2001 | 8 (13) | 15 (22) | |||
| GVHD prophylaxis | 60 | 69 | .35 | ||
| CsA + MTX ± other‡§ | 60 (100) | 68 (99) | |||
| None‡ | 0 | 1 (1) | |||
| Total no. of transplantations‖; | 60 | 69 | .12 | ||
| 1 | 53 (88) | 62 (88) | |||
| 2 | 4 (7) | 8 (12) | |||
| More than 2 | 3 (5) | 0 | |||
| Median follow-up of survivors, mo¶ | 77 (5-124) | 75 (22-123) | |||
| Variables . | Cy . | Cy + ATG . | P* . | ||
|---|---|---|---|---|---|
| N eval . | N (%) . | N eval . | N (%) . | ||
| No. of patients | 60 | 70 | |||
| No. of centers | 27 | 29 | |||
| Median age, y (range) | 60 | 26 (4-51) | 70 | 23 (1-51) | .11 |
| Age at transplantation | 60 | 70 | .70 | ||
| Younger than 10 y | 5 (8) | 9 (13) | |||
| 10-19 y | 13 (22) | 20 (28) | |||
| 20-29 y | 18 (30) | 20 (28) | |||
| 30-39 y | 13 (22) | 12 (18) | |||
| At least 40 y | 11 (18) | 9 (13) | |||
| Male sex | 60 | 35 (58) | 70 | 46 (66) | .39 |
| Karnofsky score at least 90% | 60 | 32 (53) | 67 | 41 (61) | .37 |
| Prior treatment | 60 | 69 | .99 | ||
| None | 30 (50) | 37 (53) | |||
| ATG + CsA ± other | 4 (7) | 6 (8) | |||
| ATG ± other (not CsA) | 1 (1) | 1 (2) | |||
| CsA ± other (not ATG) | 9 (15) | 11 (16) | |||
| Corticosteroids | 4 (7) | 4 (6) | |||
| Corticosteroids + other | 5 (8) | 5 (7) | |||
| Other† | 7 (12) | 6 (8) | |||
| No. of donor exposures prior to transplantation | 60 | 70 | .04 | ||
| 0 | 3 (5) | 1 (2) | |||
| 1-10 | 20 (33) | 21 (30) | |||
| More than 10 | 30 (50) | 26 (37) | |||
| Transfusions given, unknown no. | 7 (12) | 22 (31) | |||
| Median nucleated cell dose, × 108/kg (range) | 44 | 2.4 (.01-5) | 59 | 2.7 (.01-7) | .82 |
| Donor-recipient sex match | 60 | 69 | .37 | ||
| Male → Male | 21 (35) | 26 (38) | |||
| Male → Female | 15 (25) | 9 (13) | |||
| Female → Male | 14 (23) | 20 (29) | |||
| Female → Female | 10 (17) | 14 (20) | |||
| Donor-recipient CMV status | 53 | 61 | .90 | ||
| Donor +/recipient + | 28 (53) | 29 (47) | |||
| Donor +/recipient − | 10 (19) | 11 (18) | |||
| Donor −/recipient + | 5 (9) | 6 (10) | |||
| Donor −/recipient − | 10 (19) | 15 (25) | |||
| Graft type | 60 | 69 | .56 | ||
| Bone marrow | 58 (96) | 68 (99) | |||
| PBS‡C | 1 (2) | 1 (1) | |||
| BM + PBSC‡ | 1 (2) | 0 | |||
| Median time from Dx to Tx, mo (range) | 60 | 2 (<1-109) | 69 | 2 (<1-70) | .63 |
| Time from Dx to Tx | 60 | 69 | .78 | ||
| Less than 3 mo | 41 (69) | 45 (65) | |||
| 3 to less than 6 mo | 9 (15) | 8 (12) | |||
| 6-12 mo | 5 (8) | 9 (13) | |||
| At least 12 mo | 5 (8) | 7 (10) | |||
| Year of transplantation | 60 | 69 | .65 | ||
| 1994-1995 | 16 (27) | 18 (26) | |||
| 1996-1997 | 23 (38) | 22 (32) | |||
| 1998-1999 | 13 (22) | 14 (20) | |||
| 2000-2001 | 8 (13) | 15 (22) | |||
| GVHD prophylaxis | 60 | 69 | .35 | ||
| CsA + MTX ± other‡§ | 60 (100) | 68 (99) | |||
| None‡ | 0 | 1 (1) | |||
| Total no. of transplantations‖; | 60 | 69 | .12 | ||
| 1 | 53 (88) | 62 (88) | |||
| 2 | 4 (7) | 8 (12) | |||
| More than 2 | 3 (5) | 0 | |||
| Median follow-up of survivors, mo¶ | 77 (5-124) | 75 (22-123) | |||
One hundred thirty-four recipients from 38 participating transplantation centers were enrolled between 1994 and 2001. Of these, 4 patients were excluded; 2 failed to meet criteria for SAA: hepatitis B seropositive donor (n = 1) and syngeneic donor (n = 1). Among the 130 eligible patients, there were 9 minor protocol violations. These violations included the following: 3 patients received peripheral blood grafts, 4 received growth factor for hematopoietic recovery prior to day +21, 1 patient did not receive GVHD prophylaxis, and 1 patient received tacrolimus and a single dose of methotrexate for GVHD prophylaxis. Of the 130 eligible patients, 60 were randomly assigned to receive cyclophosphamide and 70 to receive cyclophosphamide and antithymocyte globulin. One patient randomly assigned to receive cyclophosphamide and antithymocyte globulin died before transplantation.
Cy indicates cyclophosphamide; eval, evaluable; CsA, cyclosporine; ±, with or without; CMV, cytomegalovirus; PBSC, peripheral blood stem cells; Dx, diagnosis; Tx, transplantation; GVHD, graft-versus-host disease; MTX, methotrexate.
The Mantel-Haenszel test was used for discrete covariates; the Kruskal-Wallis test was used for continuous covariates.
Other types of treatments were cytokines (9), androgens (2), other immune suppression (2).
Protocol violation.
Other GVHD prophylaxis were corticosteroids (10), tacrolimus with only one dose of MTX (1), corticosteroids + ATG (2), ATG (2).
All transplantations on study were the patient's first transplantation.
From date of randomization.