Wilms tumor: successes and challenges in management outside of cooperative clinical trials

Objective/background: Management of Wilms tumor [WT] in children depends on a multidisciplinary approach to treatment, and outcomes have significantly improved as reported by cooperative group clinical trials. Here, we review the clinical outcomes of patients with WT and identify challenges and barriers encountered in multidisciplinary management outside of cooperative clinical trials

Methods: We retrospectively reviewed the clinical records of 35 children with WT treated between April 2002 and June 2013 at the Children's Cancer Institute in Lebanon

Results: Upfront resection was performed in 23 cases. Biopsies were performed for Stage V tumors [n = 4], those with unresectable tumors or inferior vena caval thrombus [n = 5], and patients who had partial surgery performed elsewhere prior to presentation [n = 2]. One patient died due to toxicity prior to surgery. The tumor was Stage I in eight patients, Stage II in five patients, Stages III and IV in nine patients each, and bilateral [Stage V] in four patients. Adherence to The National Wilms Tumor Study-5 recommendations was adequate. At the time of analysis, 30 patients were free of disease and four patients had relapse-all having metastatic disease initially

Conclusion: The National Wilms Tumor Study-5 therapy resulted in favorable outcomes in children with nonmetastatic Wilms tumor in the setting of a multidisciplinary approach to therapy and resolution of financial barriers to medical care. Upstaging due to prior intervention and lung radiation therapy to all those with computed tomography-detected lung nodules may both have resulted in overtreatment of a subset of patients. Finally, the relatively high incidence of bilateral tumors suggests the need for further genetic and molecular studies in this patient population

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