Dr. Bertaina 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). She 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.

Dr. Bertaina is a well known expert in the field of allogeneic HSCT in pediatric patients affected by hematological malignancies or nonmalignant disorders. In particular, she has largely contributed to set-up the novel approach of graft manipulation based on the physical elimination of alfa/beta T cells and B cells. Dr. Bertaina has an excellent both clinical and biological expertise, as documented by the several papers that she has published in the field of pediatric hematology and oncology. Moreover, she has developed a robust expertise in the knowledge of the different aspects of immunological reconstitution of children given an allograft of hematopoietic stem cell, paying peculiar attention to the 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)
  • 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)
  • Peer Reviewed Journal Reviewer, Frontiers in Immunology (2016 - Present)
  • 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.


Stanford Advisees


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


    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


    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

  • 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


    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

  • 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


    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

  • 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


    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


    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


    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


    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


    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