Characteristics of patients enrolled in the Mayo Clinic ATG trial
| Patient no. . | Sex . | Age, y . | WHO diagnosis . | IPSS . | Date of trial enrollment* . | Treatments prior to ATG trial enrollment† . | Date of initial MDS diagnosis . | Alive or dead (specific cause of death)‡ . | Karyotype . |
|---|---|---|---|---|---|---|---|---|---|
| 1 | F | 62 | RAEB-1 | INT-1 | 4/98 | EPO, deferoxamine | 8/94 | Died 2/00 (AML) | 46,XX[30] |
| 2 | F | 74 | RA | INT-1 | 6/98 | Amifostine | 9/97 | Died 9/00 | 46,XX,del(5)(q13q33),idic(X)(p10)[13]/46, XX,del(5)(q13q33)[2]/46,XX[5] |
| 3 | F | 70 | RAEB-1 | INT-1 | 10/99 | None | 9/99 | Died 1/00 | 46,XX[30] |
| 4 | M | 63 | RAEB-1 | INT-1 | 10/01 | Thalidomide, EPO | 10/99 | Alive 9/02 | 46,XY,del(5)(q15q33),add(21)(q11.2)[8]/ 46,XY[12] |
| 5 | M | 74 | RAEB-1 | INT-2 | 11/01 | EPO | 6/01 | Alive 9/02 | 47,XY,+8[2]/46,XY[18] |
| 6 | M | 62 | RA | INT-1 | 1/02 | EPO | 2/01 | Alive 9/02 | 45,X,−Y[13]/46,XY[7] |
| 7 | F | 73 | RAEB-1 | INT-2 | 3/02 | None | 11/01 | Died 6/02 (sepsis) | 44,XX,−2,del(5)(q13q33),add(16)(q24), psu dic(19;20)(p13.3;q13.3),−21,+mar[17] |
| 8 | F | 72 | RAEB-1 | INT-2 | 5/02 | None | 9/01 | Alive 9/02 | 46,XX,+8,der(1;7)(q10;p10)[20] |
| Patient no. . | Sex . | Age, y . | WHO diagnosis . | IPSS . | Date of trial enrollment* . | Treatments prior to ATG trial enrollment† . | Date of initial MDS diagnosis . | Alive or dead (specific cause of death)‡ . | Karyotype . |
|---|---|---|---|---|---|---|---|---|---|
| 1 | F | 62 | RAEB-1 | INT-1 | 4/98 | EPO, deferoxamine | 8/94 | Died 2/00 (AML) | 46,XX[30] |
| 2 | F | 74 | RA | INT-1 | 6/98 | Amifostine | 9/97 | Died 9/00 | 46,XX,del(5)(q13q33),idic(X)(p10)[13]/46, XX,del(5)(q13q33)[2]/46,XX[5] |
| 3 | F | 70 | RAEB-1 | INT-1 | 10/99 | None | 9/99 | Died 1/00 | 46,XX[30] |
| 4 | M | 63 | RAEB-1 | INT-1 | 10/01 | Thalidomide, EPO | 10/99 | Alive 9/02 | 46,XY,del(5)(q15q33),add(21)(q11.2)[8]/ 46,XY[12] |
| 5 | M | 74 | RAEB-1 | INT-2 | 11/01 | EPO | 6/01 | Alive 9/02 | 47,XY,+8[2]/46,XY[18] |
| 6 | M | 62 | RA | INT-1 | 1/02 | EPO | 2/01 | Alive 9/02 | 45,X,−Y[13]/46,XY[7] |
| 7 | F | 73 | RAEB-1 | INT-2 | 3/02 | None | 11/01 | Died 6/02 (sepsis) | 44,XX,−2,del(5)(q13q33),add(16)(q24), psu dic(19;20)(p13.3;q13.3),−21,+mar[17] |
| 8 | F | 72 | RAEB-1 | INT-2 | 5/02 | None | 9/01 | Alive 9/02 | 46,XX,+8,der(1;7)(q10;p10)[20] |
WHO indicates World Health Organization classification of hematopoietic and lymphoid neoplasms; RA, refractory anemia; RAEB-1, refractory anemia with excess blasts; IPSS, International Prognostic Scoring System for MDS (of the International MDS Risk Analysis Workshop); INT-1 and INT-2, IPSS intermediate-1 and intermediate-2 risk categories; EPO, recombinant human erythropoietin.
The first 3 patients enrolled in this trial paid for ATG themselves. A large number of other MDS patients seen at the Mayo Clinic during the years 1998, 1999, and 2000 were also approached about participation in this trial but declined enrollment (exact records of the number of patients approached were not kept). The most common reasons patients declined enrollment were concern about the cost of the drug and administration fees and/or inability to stay in Rochester, MN, for the expected duration of treatment. In 2001, Pharmacia agreed to provide the study drug free of cost to patients, which allowed more rapid accrual of 5 remaining patients.
Not including transfusions or antimicrobial support for infections.
Patients 1, 2, 3, and 7 were followed until their deaths 22, 27, 15, and 3 months after ATG treatment, respectively. None of these patients enjoyed a hematologic improvement. Patients 4, 5, 6, and 8 are still alive and have been followed for 11, 10, 8, and 4 months, respectively; patients 6 and 8 were started on other therapies 3 months after ATG therapy (thalidomide and EPO) when no signs of improvement had been seen.