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To date, there has been a lack of pediatric experience regarding the efficacy and tolerability of immune checkpoint inhibitors after haploidentical hematopoietic stem cell transplant (HSCT). We present the case of a 22-year-old female with multiple-relapsed Hodgkin lymphoma (HL) who presented with a new relapse after haploidentical (post-haplo) HSCT. Anti-PD-1 therapy with nivolumab resulted in significant objective disease response and clinical improvement without notable side effects, including the absence of a graft-versus-host disease (GVHD). This case report suggests that immune checkpoint inhibition may be safely tolerated even in the setting of haploidentical HSCT, without triggering overt GVHD., (© 2016 Wiley Periodicals, Inc.)

There are few therapeutic options for patients with T-cell acute lymphoblastic leukemia (T-ALL) who have recurrent disease after initial matched sibling hematopoietic stem cell transplantation. While a second hematopoietic stem cell transplant (HSCT) from a haploidentical donor offers the conceptual possibility of greater graft versus leukemia effect, there is minimal literature to describe the efficacy of this approach in recurrent pediatric T-ALL. We present the case of a now 9-year-old female in whom second haploidentical HSCT, followed by successive donor lymphocyte infusions in response to minimal residual disease reemergence, has led to 3+ years of ongoing disease control without graft versus host disease and excellent quality of life., (© 2016 Wiley Periodicals, Inc.)

Background: Automated functional imaging (AFI) was introduced to two-dimensional speckle-tracking echocardiography to facilitate strain assessment in the clinical settings. In patients treated with cardiotoxic anthracyclines, AFI may be helpful in the detection of early myocardial injury when left ventricular ejection fraction (LVEF) remains normal., Methods: We retrospectively assessed feasibility of AFI in 143 echocardiograms on 102 subjects aged 0.4-22 years (mean 12.3) obtained over a 12-month period. We computed a Z-score for apical four-chamber longitudinal strain using published normal data to assess for abnormal strain in patients with and without previous exposure to anthracyclines., Results: AFI was feasible in 95.1% of echocardiograms, with low inter- and intraobserver variability. There was a statistically significant association between abnormal longitudinal strain Z-score (SZ < -2.0) and depressed LVEF (<55%, P < 0.001). However, 46% of echocardiograms with normal LVEF had abnormal SZ; half of which had no prior anthracycline exposure. The correlation between SZ and LVEF was strongest in subjects exposed to anthracyclines (r(2) = 0.12, P < 0.01). Increasing age was associated with decreasing SZ. Total cumulative dose, after adjusting for age, was inversely associated with SZ (r(2) = 0.42, P < 0.001). Time from last dose of anthracycline had no significant association with SZ., Conclusions: AFI is highly feasible in the clinical settings. The observed high prevalence of abnormal longitudinal strain in our cohort emphasizes the importance of obtaining baseline measurements prior to anthracycline treatment. The effects of anthracycline on longitudinal strain may be dose and age dependent, with younger children less likely to show abnormalities., (© 2016 Wiley Periodicals, Inc.)

Background: To compare the burden of central line-associated bloodstream infections (CLABSIs) in ambulatory versus inpatient pediatric oncology patients, and identify the epidemiology of and risk factors associated with ambulatory CLABSIs., Procedure: We prospectively identified infections and retrospectively identified central line days and characteristics associated with CLABSIs from January 2009 to October 2010. A nested case-control design was used to identify characteristics associated with ambulatory CLABSIs., Results: We identified 319 patients with central lines. There were 55 ambulatory CLABSIs during 84,705 ambulatory central line days (0.65 CLABSIs per 1,000 central line days (95% CI 0.49, 0.85)), and 19 inpatient CLABSIs during 8,682 inpatient central line days (2.2 CLABSIs per 1,000 central lines days (95% CI 1.3, 3.4)). In patients with ambulatory CLABSIs, 13% were admitted to an intensive care unit and 44% had their central lines removed due to the CLABSI. A secondary analysis with a sub-cohort, suggested children with tunneled, externalized catheters had a greater risk of ambulatory CLABSI than those with totally implantable devices (IRR 20.6, P < 0.001). Other characteristics independently associated with ambulatory CLABSIs included bone marrow transplantation within 100 days (OR 16, 95% CI 1.1, 264), previous bacteremia in any central line (OR 10, 95% CI 2.5, 43) and less than 1 month from central line insertion (OR 4.2, 95% CI 1.0, 17)., Conclusions: In pediatric oncology patients, three times more CLABSIs occur in the ambulatory than inpatient setting. Ambulatory CLABSIs carry appreciable morbidity and have identifiable, associated factors that should be addressed in future ambulatory CLABSI prevention efforts., (Copyright © 2013 Wiley Periodicals, Inc.)

We report a novel regimen for refractory post-transplant T-cell lymphoma (PTL). Our patient presented with non-Epstein-Barr virus (EBV) related, T-cell post-transplant lymphoproliferative disease (PTLD) 3.5 years after liver transplantation. Initially diagnosed as polyclonal PTLD, the disease progressed to a monoclonal, T-cell PTL that was refractory to several chemotherapy regimens but responded to a regimen consisting of fludarabine, cyclophosphamide, cytarabine, and alemtuzumab. Consolidation therapy included high-dose chemotherapy, autologous hematopoietic stem cell rescue, and radiation therapy. She remains in remission 2.5 years later. T-cell PTL is a rare disease with a poor prognosis; this regimen provides a novel, potentially curative approach for its treatment., ((c) 2007 Wiley-Liss, Inc.)

Objective: Demonstrate that high-dose cyclophosphamide (CY) is effective therapy for hepatitis-associated aplastic anemia (HAA)., Background: HAA is a sequence of seronegative hepatitis followed by aplastic anemia. Optimal treatment is matched-sibling allogeneic bone marrow transplantation (BMT). The combination of antithymocyte globulin (ATG) and cyclosporine (CSA) has also been studied, but there are scarce data regarding treatment of HAA., Procedure: Five patients (median age 14 years; range 6-17 years) with HAA and without an HLA-matched sibling were treated with high-dose CY (50 mg/kg/day IV x 4 days) followed by granulocyte-colony stimulation factor (G-CSF)., Results: After at least 1 year of follow-up, four of five patients are in remission without further immune suppression beyond high-dose CY. Of the 4 responders, median time to absolute neutrophil count (ANC) >500 microl(-1) was 51 days (range 44-369). Median time to transfusion independence for erythrocytes and platelets was 109 (range 57-679) and 160 (range 48-679) days, respectively. The fifth patient did not respond and proceeded to an unrelated donor transplant. One patient met criteria for autoimmune hepatitis (AIH) in addition to HAA. In this case, high-dose CY successfully induced remission of both diseases., Conclusions: High-dose CY induces durable remissions in HAA and may be an effective treatment for AIH., (2007 Wiley-Liss, Inc)