Browsing by Author "Locatelli, Franco"

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    Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia
    (Ferrata Storti Foundation, 2015-01) Niemeyer, Charlotte M.; Loh, Mignon L.; Cseh, Annamaria; Cooper, Todd; Dvorak, Christopher C.; Chan, Rebecca; Xicoy, Blanca; Germing, Ulrich; Kojima, Seiji; Manabe, Atsushi; Dworzak, Michael; De Moerloose, Barbara; Starý, Jan; Smith, Owen P.; Masetti, Riccardo; Catala, Albert; Bergstraesser, Eva; Ussowicz, Marek; Fabri, Oskana; Baruchel, André; Cavé, Hélène; Zwaan, Michel; Locatelli, Franco; Hasle, Henrik; van den Heuvel-Eibrink, Marry M.; Flotho, Christian; Yoshimi, Ayami; Department of Pediatrics, IU School of Medicine
    Juvenile myelomonocytic leukemia is a rare myeloproliferative disease in young children. While hematopoietic stem cell transplantation remains the only curative therapeutic option for most patients, children with juvenile myelomonocytic leukemia increasingly receive novel agents in phase I-II clinical trials as pre-transplant therapy or therapy for relapse after transplantation. However, response criteria or definitions of outcome for standardized evaluation of treatment effect in patients with juvenile myelomonocytic leukemia are currently lacking. Here we propose criteria to evaluate the response to the non-transplant therapy and definitions of remission status after hematopoietic stem cell transplantation. For the evaluation of non-transplant therapy, we defined 6 clinical variables (white blood cell count, platelet count, hematopoietic precursors and blasts in peripheral blood, bone marrow blast percentage, spleen size and extramedullary disease) and 3 genetic variables (cytogenetic, molecular and chimerism response) which serve to describe the heterogeneous picture of response to therapy in each individual case. It is hoped that these criteria will facilitate the comparison of results between clinical trials in juvenile myelomonocytic leukemia.
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    Extracorporeal membrane oxygenation in children receiving haematopoietic cell transplantation and immune effector cell therapy: an international and multidisciplinary consensus statement
    (Elsevier, 2022) Di Nardo, Matteo; Ahmad, Ali H.; Merli, Pietro; Zinter, Matthew S.; Lehman, Leslie E.; Rowan, Courtney M.; Steiner, Marie E.; Hingorani, Sangeeta; Angelo, Joseph R.; Abdel-Azim, Hisham; Khazal, Sajad J.; Shoberu, Basirat; McArthur, Jennifer; Bajwa, Rajinder; Ghafoor, Saad; Shah, Samir H.; Sandhu, Hitesh; Moody, Karen; Brown, Brandon D.; Mireles, Maria E.; Steppan, Diana; Olson, Taylor; Raman, Lakshmi; Bridges, Brian; Duncan, Christine N.; Choi, Sung Won; Swinford, Rita; Paden, Matt; Fortenberry, James D.; Peek, Giles; Tissieres, Pierre; De Luca, Daniele; Locatelli, Franco; Corbacioglu, Selim; Kneyber, Martin; Franceschini, Alessio; Nadel, Simon; Kumpf, Matthias; Loreti, Alessandra; Wösten-Van Asperen, Roelie; Gawronski, Orsola; Brierley, Joe; MacLaren, Graeme; Mahadeo, Kris M.; Pediatrics, School of Medicine
    Use of extracorporeal membrane oxygenation (ECMO) in children receiving hematopoietic cell transplantation (HCT) and/or Immune Effector Cells (IEC) remains controversial and evidence-based guidelines are lacking. Remarkable advancements in HCT and IEC therapies have changed expectations around reversibility of organ dysfunction and life-expectancy for affected patients. Herein, members of the Extracorporeal Life Support Organization (ELSO), Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network- (HCT and Cancer Immunotherapy Subgroup), the Pediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation (EBMT), the supportive care committee of the Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) and the Pediatric Intensive Care Oncology Kids in Europe Research (POKER) group of the European Society of Pediatric and Neonatal Intensive Care (ESPNIC) provide consensus recommendations on the use of ECMO in children receiving HCT-IEC. These are the first international, multi-disciplinary consensus-based recommendations on the use of ECMO in HCT-IEC pediatric patients. This manuscript may serve as a clinical decision support tool for pediatric hematologists, oncologists, and critical care physicians during the difficult decision-making process of ECMO candidacy and management. These recommendations may represent a base for future research studies focused on ECMO selection criteria and bedside management.