Outcome of allogeneic stem cell transplantation with a conditioning regimen of busulfan, cyclophosphamide and low-dose etoposide for children with myelodysplastic syndrome

Allogeneic stem cell transplantation [SCT] offers the best chance of cure and long-term survival for children with myelodysplastic syndromes [MDS]. Retrospective analysis of pediatric patients with primary MDS treated with allogeneic SCT at a single institution treated between January 1993 and December 2008. Of 16 consecutive children who received allogeneic SCT for treatment of MDS in our center, 14 patients met the criteria of MDS according WHO I and II criteria. The median age was 4.8 years [range, 1-14 years] and 64% were male. The median time from diagnosis to transplant was 6 months. MDS stage was refractory cytopenia [RC] in 9, refractory anemia with excess blasts [RAEB] in 5. Monosomy 7 was present in 35% of the patients. The majority of patients [11/14] were conditioned with a busulfan-based myeloablative [MA] regimen with addition of low-dose of etoposide [30 mg/kg]. All but one received a bone marrow graft. Nine patients achieved complete remission [CR], and seven remain alive. At a median follow-up of 3 years [range, 2-14 years] the OS and EFS was 57% [95%CI, 0.28-0.78]. Cumulative EFS at 10 years was 43% [95% CI: 0.14-0.70]. Relapse-related mortality was 21.4%; nonrelapse mortality [NRM] was 28.57%. All the survivors had etoposide in their conditioning regimen. Patients younger than 10 years had better survival [P=.001]. Children with MDS achieve encouraging OS and EFS following allogeneic SCT. A busulfan-based regimen with a lower dose of etoposide is an effective and less toxic regimen. The outcomes are best in younger patients

Cytomegalovirus infections in unrelated cord blood transplantation in pediatric patients: incidence, risk factors, and outcomes

Stem cells from umbilical cord blood [CB] have increasingly become a viable alternate source of progenitor cells for hematopoietic cell transplantation [HSCT] Cytomegalovirus [CMV] is thought to contribute significantly to HSCT morbidity and mortality. Retrospective case-control study in patients at tertiary care center. We determined the incidence, risk factors and outcomes for CMV infection and disease after unrelated cord blood transplantation [UCBT] in children. Between 2003 and 2007, 73 pediatric patients underwent UCBT and 68% of recipients were CMV seropositive. The overall incidence of CMV infection, early and late CMV infection was 58.9% [43/73], 62.8% [27/43], and 37.4% [1 6/43], respectively. In patients with early CMV infection, 6 of 27 [22%] patients progressed to develop CMV end-organ disease including pneumonitis and retinitis. High levels CMV antigenemia >70 infected cells by pp65 antigenemia assay + PMNs, P-.237 were associated with a higher risk of progression to CMV disease. The development of CMV infections was higher in CMV-seropositive recipients [P<.001] and in those who developed graft-versus-host-diseases [GVHD] [P<.001]. Other risk factors for CMV infection include the use of high-dose corticosteroids [P<.001] and older age of the recipient at the time of transplant [P<.002]. Late CMV infection was strongly associated with a previous history of early CMV infection [P<.001]. CMV infection is a significant complication in UCBT recipients in pediatric patients and is associated with an increase in transplant-related morbidity and mortality. Risk factors for CMV infections after UCBT include GVHD, use of corticosteroids, underlying diseases [hematologic malignancies] and older age. Late CMV infection was strongly associated with a previous history of CMV infection

Congenital sideroblastic anemia successfully treated with allogeneic stem cell transplantation

Therapy for patients with congenital sideroblastic anemia has been limited to blood transfusions and chelation. Five patients with Congenital Sideroblastic anemia [CSA] who were blood transfusion dependent underwent stem cell transplantation [SCT] from matched sibling donors. Their ages at SCT were 1,2,3,5 and 8 years. Conditioning consisted of cyclophasphamide 50 mg/kg/day for 4 days, busulfan 4 mg/kg/day for days and antithymocyte globulin [ATG] 30 mg/kg/for 4 doses pretransplant. For Graft vs. Host disease [GVHD] prophylaxis, cyclosporin A and methotrexate were used. All patient engrafted, and are alive with sustained engraftment and are transfusion independent. Two patients developed acute GVHD, and none of the patients developed chronic GVHD. In conclusion SCT can be curative for patients with CSA

Use of tranexamic acid in pediatric Kaposiform hemangio-endothelioma complicated with Kasabach-merritt syndrome

Kasabach-Merritt syndrome is an infrequent combination of Kaposiform hemangio-endothelioma and severe thrombocytopenia, which may be life- threatening with an overall mortality rate of 20-30% and for which there are no definitive methods of treatment. A retrospective data collection of a single institute [KFSHRC]* reporting three cases of Kasabach-Merritt syndrome, showing a very good response to tranexamic acid with the reversal of the hematological disorder, decrease in steroid requirement and diminution of the size of the lesion in few months duration. The early use of tranexamic acid in the treatment of Kasabach-Merritt syndrome, especially the non-resectable vascular tumors, was recommended to be combined with interferon-alpha or steroid therapy for children