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Myeloablative MSD-SCT for SCA: long-term outcome
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Figure 2. Rejection probability and event-free-survival (EFS) in sickle cell anemia (SCA) patients transplanted with matched-sibling donor (MSD) after myeloablative conditioning regimen. (A) Probability of rejection according to the anti-thymocyte globulin (ATG) dose. Overall cumulative incidence of rejection at five years was 20.0% (95%CI: 3.0-37.0%) in patients prepared without ATG and only 1.4% (95%CI: 0.0-3.0%) (P<0.001) in those prepared with ATG. However, the risk of rejection was not associated with the ATG dose (5-15 mg/kg vs. 20 mg/kg). (B) EFS according to the ATG dose. EFS was similar after bone marrow transplantation (BMT) and cord blood transplantation (CBT), and in patients prepared with 5-15 mg/kg ATG than in those prepared with 20 mg/kg. (C) EFS in 234 patients depending on the period of transplant. EFS improved strongly as it was only 73.3% (95%CI: 58.7-87.9%) among the 38 patients transplanted before year 2000 and 97.4% (95%CI: 95.0 -99.8%) in the 196 patients transplanted after year 2000. (D) EFS according to age over or under 15 years. EFS was similar in patients younger or older than 15 at transplant.
therapy. Thirty-two girls were pre-pubertal at transplanta- tion, and most required hormonal therapy to develop sec- ondary sexual characteristics at the bone age of 13 years. However, 9 of 32 of the pre-pubertal girls at SCT, who had reported to have spontaneously undergone normal puber- ty at the last visit, were significantly younger at transplant than those who required hormonal substitution for puber- ty induction [mean (SD) age 5.9 (2.6) vs. 10.1 (2.1); P=0.002]. At last visit, 20 females were older than 25, and four of them, who had been transplanted between 1988 and 1998 at 5.8, 6.1, 6.6 and 7.7 years of age, respectively, had had six spontaneous pregnancies and five children at approximately 20 years after SCT. Pre-transplant ovarian tissue cryopreservation has been systematically per- formed since year 1998. Unilateral oophorectomy was performed by laparoscopy under general anesthesia and ovarian cortical fragments were cryopreserved at a median age of 8.0 years (range: 3.0-26.4). Among the 93 girls trans-
planted since 1998, 11 were older than 25 years at the last visit; two wanted to become pregnant and requested auto- graft of ovarian fragments. The first of these was trans- planted at 20 years of age and had received hormone replacement therapy because of post-transplant ovarian failure. Orthotopic ovarian fragment autograft was per- formed by laparoscopy 29 months after SCT, and the first signs of recovery of ovarian function were observed nine weeks after ovarian graft. Hormone replacement therapy was stopped at four months and the patient became preg- nant six months after ovarian autograft, delivering a healthy girl at 38 weeks of gestation. She had another child three years later without requiring a new graft of ovarian fragments.31 In the second patient, transplanted at 22 years of age, autograft of ovarian fragments was per- formed 12 years post SCT, and recovery of ovarian func- tion was observed after four months, but unfortunately no pregnancy occurred thereafter.
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