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

Clinical characteristics and outcome of pediatric patients with stage IV Hodgkin lymphoma

While treatment outcomes for patients with Hodgkin lymphoma [HL] have improved remarkably, patients with disseminated disease still have a poorer outcome. Stage IV HL is often repported with other "advanced stage" categories, confusing the specific contribution of disease dissemination to the outcome. This single-institution report looks at characteristics and outcomes of this specific category. The medical records of pediatric HL patients [<14 years] from 1975 through 2003 were retrospectively reviewed and the data analyzed. Stage IV patients [n=67] had more poor -risk characteristics than patients in stages I-III [n=300] [B symptoms 86.6% vs. 19.3%, bulky disease 57.6% vs. 45.5% and mediastinal mass 77.6% vs. 29.7%; P < .001 for all characteristics]. The liver was the most common extralymphatic site [in 51.5% of patients with stage IV diseease. Stage IV patients received chemotherapy [CT] alone [n=55] or combined modality therapy [CMT] [n=12]. Fifty-four patients [80.6%] achieved complete remission, 2 [3%] partial remission, 10 [14.9%] had progressive disease and 1 was lost to follow up. Overall survival was 79.4% and event-free survival [EFS] was 63.9% at 5 years. There was a non-significant benefit for CMT [OS=91.7% v. 77.1%, P=.3; EFS=70.7% v. 62.7%, P=.3]. Ten of 12 relapsed and only 1 of 10 progressive disease patients were salvaged. On multivariate analysis, failure to achieve complete remission with CT was associated with a poorer outcome. Stage IV disease is associated with poor risk features and confers a worse outcome than stage I-III disease. Achievement of complete remission with CT is an important prognostic feature. Slow responders may require novel and/or aggressive therapy to achieve complete remission

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