Van Huynh

Successful autologous cord blood transplantation in a child with acquired severe aplastic anemia

Over 400 cases of pediatric SAA occur annually in the United States. A growing number of children with SAA may have had their stem cells harvested through cord blood collection. We describe a nine‐yr‐old male with SAA treated successfully with an autologous cord blood transplant following immunoablative chemotherapy. With the increasing number of people cryopreserving autologous cord blood, the use of autologous cord blood in the treatment of SAA might be considered as initial therapy. This case serves to discuss approaches to preparative therapy as well as the potential complications in this growing cohort of patients.

Outcome of children with multiply relapsed B-cell acute lymphoblastic leukemia: a therapeutic advances in childhood leukemia & lymphoma study

The survival of pediatric patients with multiply relapsed and/or refractory (R/R) B-cell acute lymphoblastic leukemia has historically been very poor; however, data are limited in the current era. We conducted a retrospective study to determine the outcome of multiply R/R childhood B-ALL treated at 24 TACL institutions between 2005 and 2013. Patient information, treatment, and response were collected. Prognostic factors influencing the complete remission (CR) rate and event-free survival (EFS) were analyzed. The analytic set included 578 salvage treatment attempts among 325 patients. CR rates (mean ± SE) were 51 ± 4% for patients with bone marrow R/R B-ALL who underwent a second salvage attempt, 37 ± 6% for a third attempt, and 31 ± 6% for the fourth through eighth attempts combined. For patients achieving a CR after their second, third, and fourth through eighth attempts, the 2 year EFS was 41 ± 6%, 13 ± 7%, and 27 ± 13% respectively. Our results showed slight improvement when compared to previous studies. This is the largest and most recent study to date that evaluates the outcome of this patient population. Our data will provide detailed reference for the evaluation of new agents being developed for childhood B-ALL.

Redefining treatment failure for pediatric acute leukemia in the era of minimal residual disease testing.

ABSTRACT Technologies for the detection of minimal residual disease (MRD) in leukemia and our understanding of the prognostic implications of MRD at different phases of treatment have significantly improved over the past decade. As a result, definitions of treatment failure based on bone marrow morphology by light microscopy are becoming increasingly inadequate for clinical care and trial design. In addition, novel therapies that may have increased efficacy and decreased toxicity in the setting of MRD compared to overt disease are changing clinical practice and challenging investigators to redefine treatment failure, the role of disease surveillance in remission, and clinical trial eligibility in the era of MRD.

Acute Lymphoblastic Leukemia: What Have We Learned About the Effects of This Disease and Its Treatment on the Nervous System?

The treatment of leukemia remains the success story of cancer. The patients with acute lymphoblastic leukemia survive longer and longer, with the rates of long time remission of over 80% in children and 30-40% in adults. Therefore, increased attention is given to diagnose and prevent central nervous system (CNS) complications, not only in order to increase the survival, but also in order to prevent neurologic deterioration and the resulting decreased quality of life. CNS disease is relatively rare at diagnosis, with less than 10% of the patients being diagnosed with his condition. However, the rate of CNS involvement escalates to up to 75% in the first year, if no effective brain-targeted treatments are usedwhich justifies the need for CNS prophylaxis even in the absence of frank metastatic involvement. Classically, patients received cranial radiation. However, a significant percentage of ALL survivors that received cranial radiation now present with a discouraging array of complications, including neurodevelopmental sequelae, strokes, seizures and increased rate of secondary CNS malignancies. More recent, it was suggested that effective CNS prophylaxis can be achieved with a combination of high dose systemic chemotherapy and intrathecal chemotherapy. Though less toxic, some survivors of this approach are still plagued by neurological complications, including cognitive deficits due to the effects of chemotherapy on the developing brain. More research needs to be conducted on further decreasing the rate on CNS relapse, while minimizing the therapy effects of the brain. Our knowledge of the biological mechanisms involved in radiation and chemotherapy effects of different cerebral structures has to improve. For the ALL survivors, therapies to repair the cognitive damage caused by cancer treatments are still in infancy.