Browsing by Author "Martinez, Caridad"

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    Guidelines for Pediatric Unrelated Cord Blood Transplantation-Unique Considerations.
    (Transplantation and cellular therapy, 2021-12) Dahlberg, Ann; Kurtzberg, Joanne; Boelens, Jaap; Martinez, Caridad; Carpenter, Paul; Tewari, Priti; American Society for Transplantation and Cellular Therapy Cord Blood Special Interest Group
    Cord blood (CB) is the stem cell source of choice for approximately 30% of pediatric patients undergoing hematopoietic cell transplantation. Cord blood is readily available and is a particularly appealing stem cell source for patients who lack appropriate HLA-matched related or unrelated donors. Pediatric cord blood transplant (CBT) recipients have low rates of disease relapse in the malignant setting and very low rates of chronic graft-versus-host disease (GVHD). In addition, CB has unique properties that make it the stem cell source of choice for some nonmalignant conditions such as metabolic disorders. This review provides evidence-based and experience-based pediatric-specific guidelines for CBT including considerations for infectious disease management, CB unit selection and infusion, conditioning regimen selection, and GVHD management. In addition, it covers unique bedside considerations for pediatric patients and CB banking. In concert with the other topic specific CB guidelines previously published in this series, it provides a comprehensive overview of the clinical management of pediatric CBT.
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    Immune Reconstitution and Survival of 100 SCID Patients Post Hematopoietic Cell Transplant: A PIDTC Natural History Study.
    (Blood, 2017-10-11) Heimall, Jennifer; Logan, Brent R; Cowan, Morton J; Notarangelo, Luigi D; Griffith, Linda M; Puck, Jennifer M; Kohn, Donald B; Pulsipher, Michael A; Parikh, Suhag; Martinez, Caridad; Kapoor, Neena; O'Reilly, Richard; Boyer, Michael; Pai, Sung-Yun; Goldman, Frederick; Burroughs, Lauri; Chandra, Sharat; Kletzel, Morris; Thakar, Monica; Connelly, James; Cuvelier, Geoff; Davila Saldana, Blachy J; Shereck, Evan; Knutsen, Alan; Sullivan, Kathleen E; DeSantes, Kenneth; Gillio, Alfred; Haddad, Elie; Petrovic, Aleksandra; Quigg, Troy; Smith, Angela R; Stenger, Elizabeth; Yin, Ziyan; Shearer, William T; Fleisher, Thomas; Buckley, Rebecca H; Dvorak, Christopher C
    The Primary Immune Deficiency Treatment Consortium (PIDTC) is enrolling children with severe combined immunodeficiency (SCID) to a prospective natural history study. We analyzed patients treated with allogeneic hematopoietic cell transplantation (HCT) from 2010-2014, including 68 with typical SCID and 32 with leaky SCID, Omenn Syndrome or Reticular Dysgenesis. Most (59%) were diagnosed by newborn screening or family history. The 2-year overall survival (OS) was 90%but was 95% for those infection-free at HCT vs. 81% for those with active infection (p=0.009). Other factors, including the diagnosis of typical vs. leaky SCID/Omenn Syndrome, diagnosis via family history or newborn screening (FH/NBS), use of preparative chemotherapy, or the type of donor utilized did not impact survival. While 1-year post-HCT median CD4 counts and freedom from IVIG were improved after use of preparative chemotherapy, other immunologic reconstitution parameters were not affected and the potential for late sequelae in extremely young infants requires further evaluation. Following a T-cell-replete graft, landmark analysis at Day +100 post-HCT revealed that CD3 <300 cells/uL, CD8 <50 cells/uL, CD45RA <10%, or a restricted Vβ T cell receptor repertoire (<13 of 24 families) was associated with need for second HCT or death. In the modern era, active infection continues to pose the greatest threat to survival for SCID patients. Although NBS has been effective in diagnosing SCID patients early in life, there is an urgent need to identify validated approaches through prospective trials to ensure that patients proceed to HCT infection free. The trial is registered at www.clinicaltrials.gov as NCT01186913.
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    Pulmonary Complications in Pediatric and Adolescent Patients Following Allogeneic Hematopoietic Cell Transplantation.
    (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2019-10) Broglie, Larisa; Fretham, Caitrin; Al-Seraihy, Amal; George, Biju; Kurtzberg, Joanne; Loren, Alison; MacMillan, Margaret; Martinez, Caridad; Davies, Stella M; Pasquini, Marcelo C
    Pulmonary complications after hematopoietic cell transplantation (HCT) can lead to significant morbidity and mortality. Limited evaluation of the true incidence of these complications in children and subsequent outcomes of these complications have not been evaluated recently. In April 2018, the National Heart, Lung, and Blood Institute; the Eunice Kennedy Shriver National Institute of Child Health and Human Development; and the National Cancer Institute cosponsored a meeting of experts to describe the status of pulmonary complications in children after HCT, identify critical gaps in knowledge, and explore avenues for research to advance care and optimize outcomes. The Center for International Blood and Marrow Transplant Research was used to evaluate the cumulative incidence of pulmonary complications in children and their respective survival. Of the 5022 children included in this analysis who received allogeneic HCT from 2010 to 2016, 606 developed pulmonary complications within the first year after HCT. Pneumonitis occurred in 388 patients, 125 patients developed pulmonary hemorrhage, and 200 patients had lung graft-versus-host disease (GVHD). For those developing pulmonary complications within 1 year, overall survival 100 days after diagnosis of pulmonary complications was 49% (95% confidence interval [CI], 43% to 54%) for patients with pneumonitis, 23% (95% CI, 16% to 31%) in patients with pulmonary hemorrhage, and 87% (95% CI, 81% to 91%) in patients with pulmonary GVHD. This study demonstrates the approximate incidence of these complications, as well as their significant effects on survival, and can serve as a baseline for future research.