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  1. Article ; Online: Optimizing cord blood selection.

    Ruggeri, Annalisa

    Hematology. American Society of Hematology. Education Program

    2019  Volume 2019, Issue 1, Page(s) 522–531

    Abstract: Nowadays a donor can be found for virtually all patients in need of an allogeneic stem cell transplantation, and the decision whether to use a matched or mismatched unrelated donor, an unrelated donor for umbilical cord blood transplantation (UCBT), or a ...

    Abstract Nowadays a donor can be found for virtually all patients in need of an allogeneic stem cell transplantation, and the decision whether to use a matched or mismatched unrelated donor, an unrelated donor for umbilical cord blood transplantation (UCBT), or a haploidentical donor depends not only on the availability of the donor but also on patient-, disease-, and center-related factors. This paper summarizes the recent criteria in the selection of cord blood unit, including the cell dose requirement and the HLA typing for the optimal donor choice. The main strategies to optimize the results of UCBT, the conditioning regimens, and the use of antithymocyte globulin and the other platforms of graft-versus-host disease prophylaxis are discussed. The paper describes the results of UCBT in children and adults with malignant and nonmalignant diseases and the comparative analysis with other donor type and stem cell sources. Emerging strategies, focusing on the different platforms of ex vivo expansion and the new applications using cord blood stem cell, are also examined.
    MeSH term(s) Adolescent ; Adult ; Anemia, Aplastic/blood ; Anemia, Aplastic/diagnosis ; Anemia, Aplastic/genetics ; Anemia, Aplastic/therapy ; Cord Blood Stem Cell Transplantation ; Donor Selection ; Female ; Histocompatibility Testing ; Humans ; Leukemia, Myeloid, Acute/blood ; Leukemia, Myeloid, Acute/diagnosis ; Leukemia, Myeloid, Acute/genetics ; Leukemia, Myeloid, Acute/therapy ; Male ; Transplantation Conditioning
    Language English
    Publishing date 2019-12-06
    Publishing country United States
    Document type Case Reports ; Journal Article ; Review
    ZDB-ID 2084287-9
    ISSN 1520-4383 ; 1520-4391
    ISSN (online) 1520-4383
    ISSN 1520-4391
    DOI 10.1182/hematology.2019000056
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Sirolimus-based graft-versus-host disease prophylaxis.

    Ruggeri, Annalisa

    The Lancet. Haematology

    2019  Volume 6, Issue 8, Page(s) e387–e388

    MeSH term(s) Cyclosporine ; Graft vs Host Disease ; Humans ; Mycophenolic Acid ; Sirolimus ; Stem Cell Transplantation ; Transplantation Conditioning
    Chemical Substances Cyclosporine (83HN0GTJ6D) ; Mycophenolic Acid (HU9DX48N0T) ; Sirolimus (W36ZG6FT64)
    Language English
    Publishing date 2019-06-24
    Publishing country England
    Document type Journal Article ; Comment
    ISSN 2352-3026
    ISSN (online) 2352-3026
    DOI 10.1016/S2352-3026(19)30108-5
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  3. Article ; Online: Alternative donors: cord blood for adults.

    Ruggeri, Annalisa

    Seminars in hematology

    2016  Volume 53, Issue 2, Page(s) 65–73

    Abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for patients with hematological diseases. The probability of finding a human leukocyte antigen (HLA)- identical donor among family members is around 25% and 30% that of ... ...

    Abstract Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for patients with hematological diseases. The probability of finding a human leukocyte antigen (HLA)- identical donor among family members is around 25% and 30% that of having a full matched unrelated donor in the registry. Patients in need may also benefit of a HLA-mismatched HSCT either from an haploidentical donors or from umbilical cord blood (UCB). Much has been learned about UCB transplant (UCBT) since the first human UCBT was performed back in 1988. Cord blood banks have been established worldwide for the collection, cryopreservation, and distribution of UCB for HSCT. Today, a global network of cord blood banks and transplant centers has been established with a large common inventory of more than 650,000 UCB units available, allowing for more than 40,000 UCBT worldwide in children and adults with severe hematological diseases. Several studies have been published on UCBT, assessing risk factors such as cell dose and HLA mismatch. Outcomes of several retrospective comparative studies showed similar results using other stem cell sources both in pediatric and adult setting. New strategies are ongoing to facilitate engraftment and reduce transplant-related mortality. In this issue, we review the current results of UCBT in adults with hematological malignancies and the clinical studies comparing UCBT with other transplant strategies. We provide guidelines for donor algorithm selection in UCBT setting.
    MeSH term(s) Adult ; Blood Banks ; Cord Blood Stem Cell Transplantation ; Graft Survival ; Hematologic Diseases/therapy ; Humans ; Risk Factors
    Language English
    Publishing date 2016-04
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 206923-4
    ISSN 1532-8686 ; 0037-1963
    ISSN (online) 1532-8686
    ISSN 0037-1963
    DOI 10.1053/j.seminhematol.2016.01.006
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article ; Online: Hurdles to the Adoption of Gene Therapy as a Curative Option for Transfusion-Dependent Thalassemia.

    Thuret, Isabelle / Ruggeri, Annalisa / Angelucci, Emanuele / Chabannon, Christian

    Stem cells translational medicine

    2022  Volume 11, Issue 4, Page(s) 407–414

    Abstract: Beta-thalassemia is one of the most common monogenic disorders. Standard treatment of the most severe forms, i.e., transfusion-dependent thalassemia (TDT) with long-term transfusion and iron chelation, represents a considerable medical, psychological, ... ...

    Abstract Beta-thalassemia is one of the most common monogenic disorders. Standard treatment of the most severe forms, i.e., transfusion-dependent thalassemia (TDT) with long-term transfusion and iron chelation, represents a considerable medical, psychological, and economic burden. Allogeneic hematopoietic stem cell transplantation from an HLA-identical donor is a curative treatment with excellent results in children. Recently, several gene therapy approaches were evaluated in academia or industry-sponsored clinical trials as alternative curative options for children and young adults without an HLA-identical donor. Gene therapy by addition of a functional beta-globin gene using self-inactivating lentiviral vectors in autologous stem cells resulted in transfusion independence for a majority of TDT patients across different age groups and genotypes, with a current follow-up of multiple years. More recently, promising results were reported in TDT patients treated with autologous hematopoietic stem cells edited with the clustered regularly interspaced short palindromic repeats-Cas9 technology targeting erythroid BCL11A expression, a key regulator of the normal switch from fetal to adult globin production. Patients achieved high levels of fetal hemoglobin allowing for discontinuation of transfusions. Despite remarkable clinical efficacy, 2 major hurdles to gene therapy access for TDT patients materialized in 2021: (1) a risk of secondary hematological malignancies that is complex and multifactorial in origin and not limited to the risk of insertional mutagenesis, (2) the cost-even in high-income countries-is leading to the arrest of commercialization in Europe of the first gene therapy medicinal product indicated for TDT despite conditional approval by the European Medicines Agency.
    MeSH term(s) Child ; Genetic Therapy/methods ; Hematopoietic Stem Cell Transplantation ; Humans ; Thalassemia/genetics ; Thalassemia/therapy ; Young Adult ; beta-Globins/genetics ; beta-Thalassemia/genetics ; beta-Thalassemia/therapy
    Chemical Substances beta-Globins
    Language English
    Publishing date 2022-03-10
    Publishing country England
    Document type Journal Article
    ZDB-ID 2642270-0
    ISSN 2157-6580 ; 2157-6580
    ISSN (online) 2157-6580
    ISSN 2157-6580
    DOI 10.1093/stcltm/szac007
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  5. Article ; Online: Unmanipulated haploidentical transplantation for adult patients with hematological malignancies.

    Ruggeri, Annalisa / Santoro, Nicole

    HemaSphere

    2019  Volume 3, Issue Suppl

    Language English
    Publishing date 2019-06-30
    Publishing country United States
    Document type Journal Article
    ISSN 2572-9241
    ISSN (online) 2572-9241
    DOI 10.1097/HS9.0000000000000247
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  6. Article ; Online: Haploidentical stem cell transplantation (HaploSCT) for patients with acute leukemia-an update on behalf of the ALWP of the EBMT.

    Nagler, Arnon / Ruggeri, Annalisa

    Bone marrow transplantation

    2019  Volume 54, Issue Suppl 2, Page(s) 713–718

    Abstract: Allogeneic stem cell transplantations (alloSCT) from haploidentical (>2 HLA mismatch) donors (HaploSCT) are constantly increasing in Europe. From 2005 to 2015 numbers of HaploSCT increased by close to 300%. In parallel, there is a major shift from T ... ...

    Abstract Allogeneic stem cell transplantations (alloSCT) from haploidentical (>2 HLA mismatch) donors (HaploSCT) are constantly increasing in Europe. From 2005 to 2015 numbers of HaploSCT increased by close to 300%. In parallel, there is a major shift from T deplete (CD34+ megadose) to T replete (non-T deplete) HaploSCT. Graft versus host disease (GVHD) prophylaxis also changed from CD34+ cell purifications to anti-thymocyte globulin (ATG) and lately to post-transplantation cyclophosphamide (PTCy). Novel conditioning regimens have been developed incorporating novel drugs and innovative approaches. Results are persistently improving and currently, registry-based and single-center studies showed no statistical significance difference in transplantation outcome between HaploSCT to alloSCT from unrelated donors and even from HLA-matched sibling donors, although the numbers of those studies are small and the lack of randomized studies available so far. HaploSCT have several advantages and such as the possibility to choose between different potential donors. Parameters to consider in the Haplo donor selection are age, gender, kinship, ABO blood group, CMV status, non-shared HLA Haplotypes and killer cell immunoglobulin-like receptor (KIR). Future goals are to further decrease transplant-related mortality currently mainly due to infection complications and reduce relapse rates especially in patients with high-risk acute leukemia.
    MeSH term(s) Europe ; Female ; Hematopoietic Stem Cell Transplantation/methods ; Humans ; Leukemia, Myeloid, Acute/therapy ; Male ; Transplantation Conditioning/methods ; Transplantation, Haploidentical/methods
    Language English
    Publishing date 2019-08-06
    Publishing country England
    Document type Journal Article
    ZDB-ID 632854-4
    ISSN 1476-5365 ; 0268-3369 ; 0951-3078
    ISSN (online) 1476-5365
    ISSN 0268-3369 ; 0951-3078
    DOI 10.1038/s41409-019-0610-5
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  7. Article ; Online: Trend towards reduction in COVID-19 in-hospital mortality.

    Ruggeri, Annalisa / Landoni, Giovanni / Ciceri, Fabio

    The Lancet regional health. Europe

    2021  Volume 3, Page(s) 100059

    Language English
    Publishing date 2021-02-26
    Publishing country England
    Document type Journal Article
    ISSN 2666-7762
    ISSN (online) 2666-7762
    DOI 10.1016/j.lanepe.2021.100059
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  8. Article: Cord blood transplantation for bone marrow failure syndromes: state of art.

    Pagliuca, Simona / Ruggeri, Annalisa / Peffault de Latour, Régis

    Stem cell investigation

    2019  Volume 6, Page(s) 39

    Abstract: Hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) represent the milestones of the treatment algorithm for idiopathic and inherited bone marrow failure (BMF) disorders. However, patients lacking a suitable donor or failing ...

    Abstract Hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) represent the milestones of the treatment algorithm for idiopathic and inherited bone marrow failure (BMF) disorders. However, patients lacking a suitable donor or failing IST still have a poor prognosis. Cord blood transplantation (CBT) has extended the possibility of HSCT for many patients in case of the absence of an eligible donor, and although in the last years, this procedure is less used in several hematological diseases, it remains an option for the treatment of patients with BMF syndromes. Nevertheless, optimization of conditioning regimen and cord blood unit selection is warranted to reduce the risk of graft failure and transplant-related mortality. This review summarizes the state of art of CBT in the field of BMF diseases, focusing on historical and recent issues in idiopathic aplastic anemia and inherited disorders.
    Language English
    Publishing date 2019-12-05
    Publishing country China
    Document type Journal Article ; Review
    ZDB-ID 2884645-X
    ISSN 2313-0792 ; 2306-9759
    ISSN (online) 2313-0792
    ISSN 2306-9759
    DOI 10.21037/sci.2019.10.04
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  9. Article ; Online: HLA peptide-binding pocket diversity modulates immunological complications after cord blood transplant in acute leukaemia.

    Boukouaci, Wahid / Rivera-Franco, Monica M / Volt, Fernanda / Lajnef, Mohamed / Wu, Ching-Lien / Rafii, Hanadi / Cappelli, Barbara / Scigliuolo, Graziana Maria / Kenzey, Chantal / Ruggeri, Annalisa / Rocha, Vanderson / Gluckman, Eliane / Tamouza, Ryad

    British journal of haematology

    2024  

    Abstract: Pocket motifs and their amino acid positions of HLA molecules are known to govern antigen presentation to effector cells. Our objective was to analyse their influence on the risk of graft-versus-host disease (GVHD) and relapse after umbilical cord blood ... ...

    Abstract Pocket motifs and their amino acid positions of HLA molecules are known to govern antigen presentation to effector cells. Our objective was to analyse their influence on the risk of graft-versus-host disease (GVHD) and relapse after umbilical cord blood transplant (UCBT). The transplant characteristics of 849 patients with acute leukaemia were obtained from the Eurocord/EBMT database. Higher acute (a) GVHD was associated with homozygosity of UCB HLA-C amino acid positions 77 and 80 (NN/KK) (p = 0.008). Severe aGVHD was associated with HLA-A pocket B YSAVMENVHY motif (p = 0.002) and NN and RR genotypes of the HLA-C amino acid positions 77 and 156 (p = 0.006 and p = 0.002). Such risk was also increased in case of recipient and UCB mismatches in P4 (p < 0.0001) and P9 (p = 0.003) pockets of HLA-DQB1 alleles. For chronic GVHD, the pocket B YYAVMEISNY motif of the HLA-B*15:01 allele and the absence of mismatch between recipient and UCB in the P6 pocket of HLA-DRB1 were associated with a lower risk (p = 0.0007 and p = 0.0004). In relapse, both UCB pocket B YFAVMENVHY belonging to HLA-A*32:01 and recipient pocket B YDSVGENYQY motif of the HLA-C*07:01 allele were associated with higher risk (p = 0.0026 and p = 0.015). We provide clues on HLA-mediated cellular interactions and their role in the development of GVHD and relapse.
    Language English
    Publishing date 2024-02-21
    Publishing country England
    Document type Journal Article
    ZDB-ID 80077-6
    ISSN 1365-2141 ; 0007-1048
    ISSN (online) 1365-2141
    ISSN 0007-1048
    DOI 10.1111/bjh.19339
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  10. Article ; Online: OBITUARY- Riccardo Saccardi (20th April 1956-19th February 2024).

    Bacigalupo, Andrea / Bonifazi, Francesca / Ciceri, Fabio / Gluckman, Eliane / Greco, Raffaella / Nozzoli, Chiara / Lombardini, Letizia / Martino, Massimo / Rambaldi, Alessandro / Rocha, Vanderson / Ruggeri, Annalisa / Snowden, John / Sureda, Anna

    Bone marrow transplantation

    2024  

    Language English
    Publishing date 2024-05-01
    Publishing country England
    Document type Editorial
    ZDB-ID 632854-4
    ISSN 1476-5365 ; 0268-3369 ; 0951-3078
    ISSN (online) 1476-5365
    ISSN 0268-3369 ; 0951-3078
    DOI 10.1038/s41409-024-02296-1
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