Bio

Bio


Dr. Alice Bertaina completed her MD degree at the University of Pavia in Italy, her fellowship in hematopoietic stem cell transplantation (HSCT) at the Bambino Gesù Children’s Hospital in Rome, and her PhD degree in Immunology and Biotechnology at Tor Vergata University in Rome. Until joining Stanford University in 2017, she was Head of the Stem Cell Transplant Unit in the Department of Hematology and Oncology at the Bambino Gesù Children’s Hospital in Rome (this institution currently has the largest number of children transplanted with hematopoietic progenitors/stem cells in Europe).

Dr. Bertaina is an expert in the field of allogeneic HSCT in pediatric patients affected by hematological malignancies or nonmalignant disorders. In particular, she has pioneered the novel approach of graft manipulation based on the physical elimination of alfa/beta T cells and B cells. Dr. Bertaina has excellent clinical and biological expertise, as demonstrated by her publications in the field of pediatric hematology and oncology. Moreover, she is expert in different aspects of immunological reconstitution of children given an allograft of hematopoietic stem cell, paying particular attention to innate immunity.

Academic Appointments


Administrative Appointments


  • Head of Stem Cell Transplant Unit, Department of Hematology and Oncology, Bambino Gesù Children’s Hospital Rome, Italy (2013 - 2017)
  • Physician Assistant and Researcher, Department of Hematology and Oncology, Bambino Gesù Children’s Hospital, Rome, Italy (2010 - 2013)
  • Residency in Pediatrics, Pediatric Hematology and Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy (2008 - 2010)
  • Medical and Research Fellowship, Department of Pediatrics, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy (2004 - 2008)
  • Medical Assistant, Ergonometric Service, Division of Cardiology, Fondazione IRCCS Policlinico San Matteo, Pavia (2002 - 2003)

Honors & Awards


  • Press release 'alfa/beta T-cell depleted haplo-transplant in children with acute leukemia', 55th ASH Meeting, Engineering Cell Therapies Press Program, New Orleans (December 2013)
  • Abstract Travel Award 'Identification of deregulated microRNAs in JMML', 2016 International JMML Symposium, San Diego (December 2016)
  • Invited to participate in the Annual National Ceremony for Cancer Research, Palazzo del Quirinale, By Invitation from the President of the Italian Republic (October 2015)

Boards, Advisory Committees, Professional Organizations


  • Member, Child Health Research Institute Stanford (CHRI) (2017 - Present)
  • Peer Reviewed Journal Reviewer, Frontiers in Immunology Bone Marrow Transplantation (2016 - Present)
  • Member, American Society of Hematology (2013 - Present)
  • Member, European Society for Blood and Marrow Transplantation (2010 - Present)
  • Member, Comitato per il Controllo delle Infezioni Ospedalerie (CCIO) at the OPBG, Rome (2011 - 2017)
  • Board Member, EBMT PDWP (European Society for Blood and Marrow Transplantation Pediatric Disease Working Party) (2014 - 2017)
  • Peer Reviewed Journal Reviewer, Bone Marrow Transplantation (2015 - Present)
  • Member, Associazione Italiana di Ematologia ed Oncaologia Pediatrics (2010 - Present)
  • Head, HSCT Working Party Group of AIEOP (Italian Association of Pediatric Hematology and Oncology) (2015 - 2017)
  • Member, Inborn Errors Working Group, EBMT. (2016 - Present)
  • Chair, Paediatric transplantation Session, EBMT 43 rd Annual Meeting, Marseille, France, March 26-29, 2017 (2017 - 2017)
  • Member, GITMO project entitled ‘3 Steps’, aiming at defining strategies of prophylaxis and treatment of veno-occlusive disease in transplant recipients (2016 - Present)
  • Member, Italian Group for Bone Marrow Transplantation (2017 - Present)

Professional Education


  • PhD, Tor Vergata University, Immunology and Biotechnology (2013)
  • Residency in Pediatrics, University of Pavia, Pavia, Italy, Thesis title: “NK-alloreactivity and outcome of pediatric patients with acute leukemia given an HLA-haploidentical stem cell transplantation” under Prof. Franco Locatelli supervision (2010)
  • Post-graduate fellowship, Bambino Gesù Children’s Hospital, Rome, Italy, Hematopoietic Stem Cell Transplantation (basic and applied research) (2010)
  • Medical Degree, University of Pavia, Pavia, Italy, Thesis title: “Human Metapneumovirus II in acute pediatric respiratory infection: epidemiology, clinical picture and diagnosis” (2005)

Research & Scholarship

Current Research and Scholarly Interests


Dr. Bertaina is a highly experienced clinician and will play a key role in supporting Section Chief Dr. Rajni Agarwal and Clinical Staff in the Stem Cell Transplant Unit at Lucile Packard Children’s Hospital. She will also continue her research on immune recovery and miRNA, understanding the mechanisms underlying immune reconstitution, Graft-versus-Host Disease (GvHD), and leukemia relapse after allogeneic HSCT in pediatric patients affected by hematological malignant and non-malignant disorders.

Teaching

Stanford Advisees


Publications

All Publications


  • T Cell-Depleted and T Cell-Replete HLA-Haploidentical Stem Cell Transplantation for Non-malignant Disorders CURRENT HEMATOLOGIC MALIGNANCY REPORTS Bertaina, A., Pitisci, A., Sinibaldi, M., Algeri, M. 2017; 12 (1): 68-78

    Abstract

    Hematopoietic stem cell transplantation (HSCT) is a treatment option for children with malignant and non-malignant disorders as well as an expanding number of inherited disorders. However, only a limited portion of patients in the need of an allograft have an HLA-compatible, either related or unrelated, donor. Haploidentical HSCT is now considered a valid treatment option, especially in view of the recent insights in terms of graft manipulation. This review will offer an overview of clinical results obtained through the use of haploidentical HSCT in non-malignant diseases. We will analyze major advantages and drawbacks of both T cell depleted and unmanipulated HSCT, discussing future challenges for further improving patients' outcome.T cell depletion (TCD) aims to reduce the morbidity and mortality associated with graft-versus-host disease (GvHD). However, the delayed immune recovery and the risk of graft failure still remain potential problems. In the last years, the use of post-transplant cyclophosphamide has been shown to be an alternative effective strategy to prevent GvHD in recipients of haploidentical HSCT. Recent data suggest that both T cell depleted and T cell-replete haplo-HSCT are suitable options to treat children with several types of non-malignant disorders lacking an HLA-identical donor.

    View details for DOI 10.1007/s11899-017-0364-3

    View details for Web of Science ID 000397936700009

    View details for PubMedID 28116633

  • Remestemcel-L for the treatment of graft versus host disease EXPERT REVIEW OF CLINICAL IMMUNOLOGY Locatelli, F., Algeri, M., Trevisan, V., Bertaina, A. 2017; 13 (1): 43-56

    Abstract

    Remestemcel-L, a third-party, off-the-shelf preparation of bone-marrow derived mesenchymal stromal cells (MSCs), has been developed for experimental use in acute graft-versus-host disease (aGvHD) and other immune-mediated conditions. Several preclinical and clinical studies have indeed suggested the potential of human mesenchymal stromal cells (MSCs) as an effective treatment for steroid-refractory aGvHD. However, an unambiguous demonstration of efficacy is still lacking. Areas covered: This review critically examines the biologic rationale supporting MSCs use in aGvHD and analyzes the results of published clinical trials in this setting, with a particular focus on the potential benefits and drawbacks of Remestemcel-L. For this purpose, a systematic literature search was performed in PubMed using the following keywords: 'mesenchymal stromal cells', 'mesenchymal progenitor cells', 'multipotent stromal cells', 'mesenchymal cells', 'MSC', 'Remestemcel-L', 'Prochymal', and 'graft-versus-host disease' or 'GvHD'. Expert commentary: Remestemcel-L represents a promising alternative to second-line immunosuppressive agents for the treatment of steroid-refractory aGvHD. Despite the safety and the favorable risk/benefit profile of this cell product, which has been demonstrated in several phase I-II studies, large and prospective randomized trials are required to confirm its efficacy in aGvHD and to define the optimal schedule of administration in terms of infusion timing, cell dose and pharmacological synergism.

    View details for DOI 10.1080/1744666X.2016.1208086

    View details for Web of Science ID 000390747200007

    View details for PubMedID 27399600

  • Preservation of Antigen-Specific Functions of aß T Cells and B Cells Removed from Hematopoietic Stem Cell Transplants Suggests Their Use As an Alternative Cell Source for Advanced Manipulation and Adoptive Immunotherapy. Frontiers in immunology Li Pira, G., Di Cecca, S., Biagini, S., Girolami, E., Cicchetti, E., Bertaina, V., Quintarelli, C., Caruana, I., Lucarelli, B., Merli, P., Pagliara, D., Brescia, L. P., Bertaina, A., Montanari, M., Locatelli, F. 2017; 8: 332-?

    Abstract

    Hematopoietic stem cell transplantation is standard therapy for numerous hematological diseases. The use of haploidentical donors, sharing half of the HLA alleles with the recipient, has facilitated the use of this procedure as patients can rely on availability of a haploidentical donor within their family. Since HLA disparity increases the risk of graft-versus-host disease, T-cell depletion has been used to remove alloreactive lymphocytes from the graft. Selective removal of αβ T cells, which encompass the alloreactive repertoire, combined with removal of B cells to prevent EBV-related lymphoproliferative disease, proved safe and effective in clinical studies. Depleted αβ T cells and B cells are generally discarded as by-products. Considering the possible use of donor T cells for donor lymphocyte infusions or for generation of pathogen-specific T cells as mediators of graft-versus-infection effect, we tested whether cells in the discarded fractions were functionally intact. Response to alloantigens and to viral antigens comparable to that of unmanipulated cells indicated a functional integrity of αβ T cells, in spite of the manipulation used for their depletion. Furthermore, B cells proved to be efficient antigen-presenting cells, indicating that antigen uptake, processing, and presentation were fully preserved. Therefore, we propose that separated αβ T lymphocytes could be employed for obtaining pathogen-specific T cells, applying available methods for positive selection, which eventually leads to indirect allodepletion. In addition, these functional T cells could undergo additional manipulation, such as direct allodepletion or genetic modification.

    View details for DOI 10.3389/fimmu.2017.00332

    View details for PubMedID 28386262

  • Cytomegalovirus in hematopoietic stem cell transplant recipients - management of infection EXPERT REVIEW OF HEMATOLOGY Locatelli, F., Bertaina, A., Bertaina, V., Merli, P. 2016; 9 (11): 1093-1105

    Abstract

    Cytomegalovirus (CMV) still causes significant morbidity and mortality in patients given allogeneic hematopoietic stem cell transplantation (HSCT). Despite effective pharmacotherapy, potentially life-threatening CMV disease occurs nowadays in up to 10% of HSCT recipients; moreover, routinely used anti-CMV agents have been shown to be associated with morbidity. Areas covered: This review examines different issues related to diagnosis and management of CMV infection in HSCT recipients, paying particular attention to the monitoring of CMV-specific immune recovery, approaches of adoptive cell therapy and new antiviral drugs. Expert commentary: Despite advances in diagnostic tests and treatment, there is still room for refining management of CMV in HSCT recipients. Immunological monitoring should be associated in the future to virological monitoring. The safety profile and efficacy of new anti-CMV agents should be compared with that of standard-of-care drugs. Donor-derived, pathogen-specific T cells adoptively transferred after transplantation could contribute to reduce the impact of CMV infection on patient's outcome.

    View details for DOI 10.1080/17474086.2016.1242406

    View details for Web of Science ID 000387510600010

    View details for PubMedID 27690683

  • Human NK cells: From surface receptors to clinical applications. Immunology letters Moretta, L., Pietra, G., Vacca, P., Pende, D., Moretta, F., Bertaina, A., Mingari, M. C., Locatelli, F., Moretta, A. 2016; 178: 15-19

    Abstract

    Natural killer (NK) cells play a major role in innate defenses against pathogens, primarily viruses, and are also thought to be part of the immunosurveillance against tumors. They express an array of surface receptors that mediate NK cell function. The human leukocytes antigen (HLA) class I-specific inhibitory receptors allow NK cells to detect and kill cells that have lost or under-express HLA class I antigens, a typical feature of tumor or virally infected cells. However, NK cell activation and induction of cytolytic activity and cytokine production depends on another important checkpoint, namely the expression on target cells of ligands recognized by activating NK receptors. Despite their potent cytolytic activity, NK cells frequently fail to eliminate tumors. This is due to mechanisms of tumor escape, determined by the tumor cells themselves or by tumor-associated cells (i.e. the tumor microenvironment) via the release of soluble suppressive factors or the induction of inhibitory loops involving induction of regulatory T cells, M2-polarized macrophages and myeloid-derived suppressor cells. The most important clinical application involving NK cells is the cure of high-risk leukemias in the haplo-identical hematopoietic stem cell transplant (HSCT) setting. NK cells originated from hematopoietic stem cells (HSC) of HLA-haploidentical donors may express Killer Immunoglobulin-like receptors (KIRs) that are mismatched with the HLA class I alleles of the recipient. This allows NK cells to kill leukemia blasts residual after the conditioning regimen, while sparing normal cells (that do not express ligands for activating NK receptors). More recent approaches based on the specific removal of TCR α/β(+) T cells and of CD19(+) B cells, allow the infusion, together with CD34(+) HSC, of mature KIR(+) NK cells and of TCR γ/δ(+) T cells, both characterized by a potent anti-leukemia activity. This greatly reduces the time interval necessary to obtain alloreactive, KIR(+) NK cells derived from donor HSC. Another promising approach is based on the use of anti-KIR blocking monoclonal antibodies (mAbs), rendering alloreactive any KIR(+) NK cells.

    View details for DOI 10.1016/j.imlet.2016.05.007

    View details for PubMedID 27185471

  • Accuracy of Bedside Paediatric Early Warning System (BedsidePEWS) in a Pediatric Stem Cell Transplant Unit. Journal of pediatric oncology nursing Gawronski, O., Ciofi degli Atti, M. L., Di Ciommo, V., Cecchetti, C., Bertaina, A., Tiozzo, E., Raponi, M. 2016; 33 (4): 249-256

    Abstract

    Hospital mortality in children who undergo stem cell transplant (SCT) is high. Early warning scores aim at identifying deteriorating patients and at preventing adverse outcomes. The bedside pediatric early warning system (BedsidePEWS) is a pediatric early warning score based on 7 clinical indicators, ranging from 0 (all indicators within normal ranges for age) to 26. The aim of this case-control study was to assess the performance of BedsidePEWS in identifying clinical deterioration events among children admitted to an SCT unit. Cases were defined as clinical deterioration events; controls were all the other patients hospitalized on the same ward at the time of case occurrence. BedsidePEWS was retrospectively measured at 4-hour intervals in cases and controls 24 hours before an event (T4-T24). We studied 19 cases and 80 controls. The score significantly increased in cases from a median of 4 at T24 to a median of 14 at T4. The proportion of correctly classified cases and controls was >90% since T8. The area under the curve receiver operating characteristic was 0.9. BedsidePEWS is an accurate screening tool to predict clinical deterioration in SCT patients.

    View details for DOI 10.1177/1043454215600154

    View details for PubMedID 26497915

  • Hematopoietic stem cell transplantation: Improving alloreactive Bw4 donor selection by genotyping codon 86 of KIR3DL1/S1. European journal of immunology Alicata, C., Pende, D., Meazza, R., Canevali, P., Loiacono, F., Bertaina, A., Locatelli, F., Nemat-Gorgani, N., Guethlein, L. A., Parham, P., Moretta, L., Moretta, A., Bottino, C., Norman, P. J., Falco, M. 2016; 46 (6): 1511-1517

    Abstract

    KIR3DL1 is a natural killer (NK) cell receptor that recognizes the Bw4 epitope of human leukocyte antigen (HLA) class I molecules. Following hematopoietic stem cell transplantation for patients lacking Bw4, KIR3DL1-expressing NK cells from Bw4-positive donors can be alloreactive and eliminate tumor cells. However, KIR3DL1 alleles having T instead of C at nucleotide 320 (encoding leucine 86 instead of serine 86) are not expressed on the cell surface. Thus, not all individuals testing positive for KIR3DL1 are optimal donors for Bw4-negative recipients. Therefore, we developed a method for genotyping codon 86, which was validated by its perfect correlation with NK cell phenotype for 100 donors of diverse KIR3DL1/S1 genotype. We typed 600 donors and found that ∼12.2% had the KIR3DL1 gene, but did not express cell-surface KIR3DL1. By contrast, high-expressing allotypes were identified when haplotypes from four families with duplicated KIR3DL1/S1 genes were characterized at high resolution. Identifying donors who have KIR3DL1 but lack cell-surface KIR3DL1 would refine donor selection. With this technique, the number of individuals identified who may not be optimal donors for Bw4-negative patients increases by threefold, when compared with standard methods. Taken together, we propose that allele typing of killer cell Ig-like receptor (KIR) polymorphisms should become a standard practice when selecting donors.

    View details for DOI 10.1002/eji.201546236

    View details for PubMedID 26990677

  • ?d T-cell reconstitution after HLA-haploidentical hematopoietic transplantation depleted of TCR-aß+/CD19+ lymphocytes. Blood Airoldi, I., Bertaina, A., Prigione, I., Zorzoli, A., Pagliara, D., Cocco, C., Meazza, R., Loiacono, F., Lucarelli, B., Bernardo, M. E., Barbarito, G., Pende, D., Moretta, A., Pistoia, V., Moretta, L., Locatelli, F. 2015; 125 (15): 2349-2358

    Abstract

    We prospectively assessed functional and phenotypic characteristics of γδ T lymphocytes up to 7 months after HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) depleted of αβ(+) T cells and CD19(+) B cells in 27 children with either malignant or nonmalignant disorders. We demonstrate that (1) γδ T cells are the predominant T-cell population in patients during the first weeks after transplantation, being mainly, albeit not only, derived from cells infused with the graft and expanding in vivo; (2) central-memory cells predominated very early posttransplantation for both Vδ1 and Vδ2 subsets; (3) Vδ1 cells are specifically expanded in patients experiencing cytomegalovirus reactivation and are more cytotoxic compared with those of children who did not experience reactivation; (4) these subsets display a cytotoxic phenotype and degranulate when challenged with primary acute myeloid and lymphoid leukemia blasts; and (5) Vδ2 cells are expanded in vitro after exposure to zoledronic acid (ZOL) and efficiently lyse primary lymphoid and myeloid blasts. This is the first detailed characterization of γδ T cells emerging in peripheral blood of children after CD19(+) B-cell and αβ(+) T-cell-depleted haplo-HSCT. Our results can be instrumental to the development of clinical trials using ZOL for improving γδ T-cell killing capacity against leukemia cells. This trial was registered at www.clinicaltrials.gov as #NCT01810120.

    View details for DOI 10.1182/blood-2014-09-599423

    View details for PubMedID 25612623

  • Negative depletion of alpha/beta(+) T cells and of CD19+B lymphocytes: A novel frontier to optimize the effect of innate immunity in HLA-mismatched hematopoietic stem cell transplantation IMMUNOLOGY LETTERS Locatelli, F., Bauquet, A., Palumbo, G., Moretta, F., Bertaina, A. 2013; 155 (1-2): 21-23

    Abstract

    In recent years, infusion of T-cell depleted hematopoietic stem cells from an HLA-haploidentical relative has been shown to represent a suitable and effective, alternative option in patients in need of an allograft who lack an HLA-identical relative. In particular, this type of allograft is associated with the enormous advantage of offering an immediate transplant treatment to virtually all pediatric patients without an HLA-matched donor, whether related or unrelated, or a suitable umbilical cord blood unit. Several studies have shown that in patients given a T-cell depleted transplant relevant part of the anti-leukemia effect is mediated by alloreactive (i.e. KIR/HLA mismatched) Natural Killer cells originated from donor hematopoietic stem cells. After infusion of positively selected hematopoietic stem cell, fully functioning Natural Killer cells emerge in the recipient peripheral blood, persisting over time, only several weeks after the allograft. We have developed a new method of T-cell depletion (based on the physical elimination of mature T cells carrying α and β chains of the T-cell receptor), which permits to maintain mature donor-derived alloreactive Natural Killer cells and γδ(+) T cells in the graft. We, thus, started a formal study in children with hematological disorders aimed at evaluating the safety and efficacy of this approach. Preliminary results on 60 children transplanted so far after this type of graft manipulation are particularly promising.

    View details for DOI 10.1016/j.imlet.2013.09.027

    View details for Web of Science ID 000328178600007

    View details for PubMedID 24091162

  • Strategies to optimize the outcome of children given T-cell depleted HLA-haploidentical hematopoietic stem cell transplantation BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY Locatelli, F., Vinti, L., Palumbo, G., Rossi, F., Bertaina, A., Mastronuzzi, A., Bernardo, M. E., Rutella, S., Dellabona, P., Giorgiani, G., Moretta, A., Moretta, L. 2011; 24 (3): 339-349

    Abstract

    The most advanced frontier of allogeneic hematopoietic stem cell transplantation (allo-HSCT) is represented by the use of an HLA-partially matched relative as donor. In this type of transplantation, donor-derived natural killer (NK) cells, which are alloreactive toward recipient cells, significantly contribute to the eradication of leukemia blasts. Alloreactive NK cells may also kill host dendritic cells and T lymphocytes, thus preventing graft-versus-host disease and graft rejection, respectively. Sophisticated strategies of adoptive infusion of T-cell lines/clones specific for the most life-threatening pathogens (namely cytomegalovirus, Epstein-Barr virus, Aspergillus and Adenovirus) have been envisaged, and successfully tested in a few pilot trials, to protect the recipient in the early post-transplantation period. In these patients, also ex-vivo expanded mesenchymal stromal cells have been shown to be beneficial for preventing graft failure. Novel and effective strategies aimed at further augmenting the graft-versus-leukemia effect and at optimizing prevention/treatment of opportunistic/viral infections are warranted.

    View details for DOI 10.1016/j.beha.2011.04.004

    View details for Web of Science ID 000295655400004

    View details for PubMedID 21925087

  • Reconstitution of repertoire of natural killer cell receptors after transplantation: just a question of time? BONE MARROW TRANSPLANTATION Locatelli, F., Bertaina, A. 2010; 45 (6): 968-969

    View details for DOI 10.1038/bmt.2010.116

    View details for Web of Science ID 000278573600002

    View details for PubMedID 20531444

  • Cord blood transplantation in patients with hemoglobinopathies TRANSFUSION AND APHERESIS SCIENCE Boncimino, A., Bertaina, A., Locatelli, F. 2010; 42 (3): 277-281

    Abstract

    Despite the optimization of conventional treatment, both thalassemia and sickle cell disease are still associated with significant morbidity and mortality, especially in developing countries. Allogeneic transplantation of hematopoietic progenitors is the only curative treatment and represents an attractive option for these patients. In view of the low incidence of graft-versus-host disease associated with the procedure, allogeneic cord blood transplantation (CBT) is particularly appealing for patients with non-malignant disorders. Available evidence indicates that related donor CBT is a safe and effective option for patients with hemoglobinopathies, able to offer results at least as good as those reported using bone marrow cells.

    View details for DOI 10.1016/j.transci.2010.03.006

    View details for Web of Science ID 000279531900010

    View details for PubMedID 20382570

  • The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation COOLEY'S ANEMIA: NINTH SYMPOSIUM Bertaina, A., Bernardo, M. E., Mastronuzzi, A., La Nasa, G., Locatelli, F. 2010; 1202: 141-148

    Abstract

    Allogeneic hematopoietic stem cell transplantation (HSCT) still remains the only curative treatment for patients with thalassemia major (TM). However, HSCT is associated with a non-negligible risk of both transplantation-related mortality (TRM) and morbidity. Great interest and relevant expectations have been raised by the introduction in the clinical practice of reduced-intensity preparative regimens, which may represent an effective strategy to reduce the toxicity of transplantation and may also help reduce the incidence of late effects. Although some reports have documented the feasibility of using reduced-intensity preparative regimens for successfully treating patients with TM, a high incidence of graft failure has been frequently reported. Recently, treosulfan-based myeloablation has been demonstrated to be associated with limited extra-medullary toxicity and a high rate of sustained donor engraftment. This novel approach is a promising alternative for reducing the risk of life-threatening complications and increasing the number of TM patients successfully cured with an allograft.

    View details for DOI 10.1111/j.1749-6632.2010.05590.x

    View details for Web of Science ID 000283099600022

    View details for PubMedID 20712785

  • Transplantation and innate immunity: the lesson of natural killer cells ITALIAN JOURNAL OF PEDIATRICS Bertaina, A., Locatelli, F., Moretta, L. 2009; 35

    Abstract

    Natural killer cells have been demonstrated to play a major role in mediating an anti-leukemia effect in patients given a T-cell depleted allogeneic hematopoietic stem cell transplantation from an HLA-haploidentical family donor. In particular, donor-derived natural killer cells, which are alloreactive (i.e. KIR/HLA mismatched) towards recipient cells, significantly contribute to the eradication of leukemia blasts escaping the preparative regimen to transplantation. A recent study on high-risk pediatric acute lymphoblastic leukemia refractory to chemotherapy further highlighted the importance of donors with alloreactive natural killer cells in haploidentical hematopoietic stem cell transplantation, as it demonstrated that these cells can emerge starting from the fourth-fifth month after the allograft and persist for many months. This study represents a major breakthrough in the cure of otherwise fatal leukemias, providing information on the best criteria for choosing the optimal donor.

    View details for DOI 10.1186/1824-7288-35-44

    View details for Web of Science ID 000208014800043

    View details for PubMedID 20076779

    View details for PubMedCentralID PMC2806872