Gal Goldstein

Factor V Leiden and Antiphospholipid Antibodies in Either Mothers or Infants Increase the Risk for Perinatal Arterial Ischemic Stroke

Background and Purpose— The objective was to investigate the role of infant and maternal thrombophilia in a cohort of mothers and infants presenting with perinatal arterial ischemic stroke. Methods— Forty-seven infants with clinically and radiologically confirmed perinatal arterial ischemic stroke underwent thrombophilia workup: factor V Leiden (FVL), PII20210A mutation, Methylene-tetrahydrofolate reductase 677T polymorphism, protein C, protein S, antithrombin, FVIII, and antiphospholipid antibodies. Thrombophilia data were available for 23 mother–infant pairs and compared with control populations to evaluate the risk for PAS. Results— Thirty of 47 (64%) infants and 15 of 22 mothers (68%) had evidence of thrombophilia. In 18 of 23 (78%) mother–infant pairs, there was at least 1 thrombophilic risk factor, but 15 pairs were mismatched in pathology. Among infants, FVL, protein C deficiency, and presence of antiphospholipid antibodies prevailed (OR, 4.2; 95% CI, 1.5–11.3; OR, 12.2; 95% CI, 2.5–59.9; OR, 4.1; 95% CI, 1.4–12.2, respectively). Interestingly FVL prevailed in almost one-third of mothers (OR, 8.5; 95% CI, 4.1–17.5) and 18% of mothers had antiphospholipid antibodies (OR, 3.8l; 95% CI, 1.5–10.0). Conclusions— Maternal and neonatal thrombophilia, especially presence of FVL or antiphospholipid antibodies, may be important in the pathogenesis of perinatal arterial ischemic stroke. The nature of thrombophilic mother–infant risk potential interactions warrants further investigation.

Umbilical cord blood transplantation: Pros, cons and beyond

Large body of clinical and scientific data has been generated since the first cord blood transplantation (CBT) was performed in 1989. Superior immune plasticity of CB grafts, that allows for less stringent HLA matching, is especially valuable in the face of a persistently growing need for unrelated donor (UD) transplants. Limited cell dose remains the main setback of CBT, particularly in adult population. New strategies, such as transplantation with two cord blood units or using non-myeloablative conditioning, have remarkably expanded the availability of CB transplants in adults with hematological malignancies. Clinical trials with in vitro expanded CB-derived stem cells are under way. Currently cord blood is considered a second best choice after matched bone marrow. However, results of recent international studies indicate that in particular clinical settings, such as in children with leukemia, CB may become a frontline hematopoietic stem cell (HSC) source for transplantation. Recent advances in understanding the unique biology of cord blood will further expand indications for its use in different settings, including those beyond hematopoietic stem cells transplantation (HSCT).

Risk Factors for Central Venous Catheter Thrombotic Complications in Children and Adolescents With Cancer

The use of central venous catheters (CVCs) has greatly improved the quality of care in children with cancer, yet these catheters may cause serious infectious and thrombotic complications. The aim of this prospective registry study was to assess the host and CVC‐related risk factors for CVC‐created thrombotic complications.

Impact of conditioning on outcome of hematopoietic stem cell transplantation for wiskott-Aldrich syndrome.

Hematopoietic stem cell transplantation (HSCT) is the treatment of choice for Wiskott-Aldrich syndrome (WAS). The aim of this retrospective study is to report the effect of the conditioning regimen and donor source on disease-free survival (DFS) in children undergoing HSCT for WAS. Fourteen children who underwent HSCT at 4 Israeli centers from 1996 to 2011 were included in this study. Five children were transplanted from matched related donors (4/5 siblings, 1/5 fully matched uncle) and other donors were used in 9 children. Six patients were conditioned with full dose busulfan/cyclophosphamide (Bu/Cy) whereas 8 patients were conditioned with other regimens. Thirteen of 14 patients (92.8%) are alive with a median follow-up of 3.4 years (range, 5 mo to 12.5 y). Nine patients (64.3%) survive with complete clinical, immunologic, and hematologic recovery. Children conditioned with full dose Bu/Cy had a 100% DFS, compared with children conditioned with other regimens, 25%±19% (P=0.022). Donor source was not associated with DFS. Graft failure was related to the use of conditioning regimens other than full dose Bu/Cy and not to the donor source. Further studies are required to determine the best conditioning regimen and optimal donor source for children with WAS.

Pulmonary function studies in children treated by chemoradiotherapy and stem cell transplantation

Management of pediatric patients with malignant and hematological diseases is frequently associated with pulmonary complications. We assessed pulmonary function at diagnosis and during a 5‐year follow‐up to identify risk factors associated with pulmonary deterioration.

The role of immunoglobulin prophylaxis for prevention of cytomegalovirus infection in pediatric hematopoietic stem cell transplantation recipients.

Following cessation of intravenous immunoglobulin (IVIg) administration for allogeneic hematopoietic stem cell transplantation (HSCT) recipients at our unit, we observed a sharp decline in the incidence of cytomegalovirus (CMV) infection.

Do immunocompromised children benefit from having surgical lung biopsy performed

Background: Surgical lung biopsy is considered a gold standard for the evaluation of pulmonary disease in immunocompromised children. However, in the literature, its accuracy and the rate of complications vary. Objective: We aimed to evaluate the yield of surgical lung biopsies in the management of persistent pulmonary findings in immunocompromised children. Methods: We performed a retrospective review of clinical records of immunocompromised children who underwent surgical lung biopsies, and evaluated the impact that preoperative factors had on outcomes. Results: Twenty-five patients underwent 27 surgical lung biopsies. The underlying immunodeficiency included allogeneic stem cell transplantation (n = 12), chemotherapy for solid tumors (n = 6), hematologic malignancy (n = 4), primary immunodeficiency (n = 4) and chronic steroid use (n = 1). Biopsies provided a specific histopathologic or microbiologic diagnosis in 10 cases (37%). No preoperative factor predicted a diagnostic biopsy. Five of the 27 biopsies were beneficial for the patients (18%). A major complication related to the procedure was reported for 1 biopsy (4%). Conclusions: We conclude that surgical lung biopsy in pediatric immunocompromised patients appears to be safe, but has a relatively low diagnostic yield and an even lower yield with regards to the benefit it provides.

Abnormal body mass index at diagnosis in patients with Ewing sarcoma is associated with inferior tumor necrosis.

Abnormal body mass index (BMI) in cancer patients at diagnosis has been associated with lower survival rates. The degree of tumor necrosis after induction chemotherapy in Ewing sarcoma (EWS) is highly associated with treatment failure. We analyzed the effect of BMI on tumor necrosis in children and young adults undergoing induction treatment for EWS.