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  1. Article ; Online: The CAR T-cell race: The rules of the game.

    Balduzzi, Adriana

    British journal of haematology

    2024  

    Abstract: The management of paediatric patients with refractory acute lymphoblastic leukaemia eligible for the CAR T cell product tisagenlecleucel involves multiple decision points between the process of patient referral to product infusion. How to address the ... ...

    Abstract The management of paediatric patients with refractory acute lymphoblastic leukaemia eligible for the CAR T cell product tisagenlecleucel involves multiple decision points between the process of patient referral to product infusion. How to address the individual patient's circumstances, optimize apheresis yields and, above all, plan the best bridging chemotherapy is clearly detailed in these comprehensive and practical recommendations by Kumar Mishra and colleagues. Commentary on: Mishra et al. Practice guideline: Preparation for CAR T-cell therapy in children and young adults with B-acute lymphoblastic leukaemia. Br J Haematol 2024 (Online ahead of print). doi: 10.1111/bjh.19381.
    Language English
    Publishing date 2024-04-08
    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.19452
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  2. Article ; Online: Immunological response after mild COVID-19: How long will it last?

    Balduzzi, Adriana

    EBioMedicine

    2021  Volume 72, Page(s) 103597

    MeSH term(s) COVID-19 ; Humans ; SARS-CoV-2 ; Severity of Illness Index
    Language English
    Publishing date 2021-09-24
    Publishing country Netherlands
    Document type Journal Article ; Comment
    ZDB-ID 2851331-9
    ISSN 2352-3964
    ISSN (online) 2352-3964
    DOI 10.1016/j.ebiom.2021.103597
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article ; Online: Immunological response after mild COVID-19

    Adriana Balduzzi

    EBioMedicine, Vol 72, Iss , Pp 103597- (2021)

    How long will it last?

    2021  

    Keywords Medicine ; R ; Medicine (General) ; R5-920
    Language English
    Publishing date 2021-10-01T00:00:00Z
    Publisher Elsevier
    Document type Article ; Online
    Database BASE - Bielefeld Academic Search Engine (life sciences selection)

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  4. Article ; Online: The Value of Minimal Residual Disease (and Diamonds).

    Balduzzi, Adriana

    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

    2017  Volume 23, Issue 1, Page(s) 3–5

    Language English
    Publishing date 2017-01
    Publishing country United States
    Document type Journal Article
    ZDB-ID 1474865-4
    ISSN 1523-6536 ; 1083-8791
    ISSN (online) 1523-6536
    ISSN 1083-8791
    DOI 10.1016/j.bbmt.2016.11.012
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  5. Article: Editorial: Allogeneic haematopoietic stem cell transplantation for children with acute lymphoblastic leukaemia in the era of immunotherapy.

    Peters, Christina / Balduzzi, Adriana / Bader, Peter

    Frontiers in pediatrics

    2022  Volume 10, Page(s) 959471

    Language English
    Publishing date 2022-08-16
    Publishing country Switzerland
    Document type Editorial
    ZDB-ID 2711999-3
    ISSN 2296-2360
    ISSN 2296-2360
    DOI 10.3389/fped.2022.959471
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  6. Article: Acute Lymphoblastic Leukaemia in the Youngest: Haematopoietic Stem Cell Transplantation and Beyond.

    Balduzzi, Adriana / Buechner, Jochen / Ifversen, Marianne / Dalle, Jean-Hugues / Colita, Anca M / Bierings, Marc

    Frontiers in pediatrics

    2022  Volume 10, Page(s) 807992

    Abstract: The ALL SCTped 2012 FORUM (For Omitting Radiation Under Majority age) trial compared outcomes for children ≥4 years of age transplanted for acute lymphoblastic leukaemia (ALL) who were randomised to myeloablation with a total body irradiation (TBI)-based ...

    Abstract The ALL SCTped 2012 FORUM (For Omitting Radiation Under Majority age) trial compared outcomes for children ≥4 years of age transplanted for acute lymphoblastic leukaemia (ALL) who were randomised to myeloablation with a total body irradiation (TBI)-based or chemotherapy-based conditioning regimen. The TBI-based preparation was associated with a lower rate of relapse compared with chemoconditioning. Nevertheless, the age considered suitable for TBI was progressively raised over time to spare the most fragile youngest patients from irradiation-related complications. The best approach to use for children <4 years of age remains unclear. Children diagnosed with ALL in their first year of life, defined as infants, have a remarkably poorer prognosis compared with older children. This is largely explained by the biology of their ALL, with infants often carrying a
    Language English
    Publishing date 2022-02-24
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2711999-3
    ISSN 2296-2360
    ISSN 2296-2360
    DOI 10.3389/fped.2022.807992
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  7. Article ; Online: A novel flow-cytometric based method to assess post-HSCT donor chimerism exploiting RNA hybridization.

    Nucera, Silvia / Sindoni, Marco M / Bugarin, Cristina / Villa, Tiziana / Biondi, Andrea / Balduzzi, Adriana / Gaipa, Giuseppe

    Bone marrow transplantation

    2023  Volume 59, Issue 2, Page(s) 171–177

    Abstract: Analysis of donor-recipient chimerism after hematopoietic stem cell transplantation (HSCT) is of pivotal importance for patient's clinical management, especially in the context of mixed chimerism. Patients are routinely monitored for chimerism in sorted ... ...

    Abstract Analysis of donor-recipient chimerism after hematopoietic stem cell transplantation (HSCT) is of pivotal importance for patient's clinical management, especially in the context of mixed chimerism. Patients are routinely monitored for chimerism in sorted subsets of peripheral blood cells. However, measurement of chimerism in sorted immune cell subsets is technically challenging and time consuming. We here propose a novel, flow cytometry-based approach to detect donor cell chimerism in sex-mismatched HSCT. We exploit RNA PrimeFlow™ system, based on RNA hybridization, to detect mRNA from a lysine demethylase encoded by Y chromosome, KDM5D. This approach allows to distinguish male and female derived cells with around 1% sensitivity. The procedure can be coupled with multiparametric immunophenotyping to assess chimerism in specific immune cell subsets without the need for prior FACS-sorting. We apply this method to a cohort of HSCT patients (n = 10) and we show that it is consistent with standard PCR-based method. We also show that different T lymphocyte subsets display variable degrees of donor chimerism, especially in CD8+ T cell compartment where we observe an enrichment for recipient chimerism in central memory T cells. This method can be exploited to advance current knowledge on immune reconstitution focusing on specific subsets avoiding prior FACS-sorting.
    MeSH term(s) Humans ; Male ; Female ; Chimerism ; Flow Cytometry ; RNA ; Hematopoietic Stem Cell Transplantation/methods ; T-Lymphocyte Subsets ; Transplantation Chimera ; Minor Histocompatibility Antigens ; Histone Demethylases/genetics
    Chemical Substances RNA (63231-63-0) ; KDM5D protein, human (EC 1.14.11.-) ; Minor Histocompatibility Antigens ; Histone Demethylases (EC 1.14.11.-)
    Language English
    Publishing date 2023-11-07
    Publishing country England
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 632854-4
    ISSN 1476-5365 ; 0268-3369 ; 0951-3078
    ISSN (online) 1476-5365
    ISSN 0268-3369 ; 0951-3078
    DOI 10.1038/s41409-023-02143-9
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    In stock of ZB MED Cologne/Königswinter

    Zs.A 2168: Show issues Location:
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  8. Article ; Online: Long-Term Complications after Allogeneic Hematopoietic Stem Cell Transplantation with Treosulfan- or Busulfan-Based Conditioning in Pediatric Patients with Acute Leukemia or Myelodysplastic Syndrome: Results of an Associazione Italiana Ematologia Oncologia Pediatrica Retrospective Study.

    Saglio, Francesco / Pagliara, Daria / Zecca, Marco / Balduzzi, Adriana / Cattoni, Alessandro / Prete, Arcangelo / Tambaro, Francesco Paolo / Faraci, Maura / Calore, Elisabetta / Locatelli, Franco / Fagioli, Franca

    Transplantation and cellular therapy

    2024  Volume 30, Issue 4, Page(s) 433.e1–433.e10

    Abstract: Patients undergoing hematopoietic stem cell transplantation (HSCT) for hematologic malignancies during childhood have an increased risk of developing long-term sequelae that are in part attributable to the conditioning regimen. The present study aimed to ...

    Abstract Patients undergoing hematopoietic stem cell transplantation (HSCT) for hematologic malignancies during childhood have an increased risk of developing long-term sequelae that are in part attributable to the conditioning regimen. The present study aimed to assess the occurrence of long-term toxicities in a population of children who underwent HSCT for hematologic malignancies using either treosulfan or busulfan in the conditioning regimen. The cumulative incidences of growth impairment, altered gonadal function, altered thyroid function, cataracts, secondary malignant neoplasia, and altered pulmonary function were evaluated retrospectively by univariable and multivariable analyses in a population of 521 pediatric patients with acute leukemias or myelodysplastic syndromes treated in 20 Italian transplant centers affiliated with the Associazione Italiana Ematologia ed Oncologia Pediatrica (AIEOP). The median duration of follow-up for the entire study population was 7.1 years (range, 1 to 16 years). Overall, a larger proportion of patients given busulfan developed long-term toxicities compared to patients treated with treosulfan (34% versus 20%; P = .01). In univariable analysis, gonadal toxicity developed in 10% of patients who received treosulfan (95% confidence interval [CI], 3% to 15%), compared with 38% (95% CI, 24% to 39%) of busulfan-treated patients (P = .02), and this finding was confirmed by multivariable analysis (relative risk, .51; 95% CI, .34 to .76; P = .0009). We did not find any statistically significant associations between the occurrence of other long-term toxicities and the use of either busulfan or treosulfan. This study provides evidence that the use of treosulfan is correlated with a reduced incidence of gonadal toxicity in children undergoing HSCT for hematologic malignancies.
    MeSH term(s) Humans ; Child ; Busulfan/adverse effects ; Busulfan/analogs & derivatives ; Retrospective Studies ; Transplantation Conditioning/adverse effects ; Transplantation Conditioning/methods ; Hematopoietic Stem Cell Transplantation/adverse effects ; Hematopoietic Stem Cell Transplantation/methods ; Leukemia, Myeloid, Acute/therapy ; Myelodysplastic Syndromes/therapy ; Hematologic Neoplasms
    Chemical Substances treosulfan (CO61ER3EPI) ; Busulfan (G1LN9045DK)
    Language English
    Publishing date 2024-01-02
    Publishing country United States
    Document type Journal Article
    ZDB-ID 3062231-1
    ISSN 2666-6367
    ISSN (online) 2666-6367
    DOI 10.1016/j.jtct.2023.12.671
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    Zs.A 5082: Show issues Location:
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  9. Article ; Online: A Review of Romiplostim Mechanism of Action and Clinical Applicability.

    Bussel, James B / Soff, Gerald / Balduzzi, Adriana / Cooper, Nichola / Lawrence, Tatiana / Semple, John W

    Drug design, development and therapy

    2021  Volume 15, Page(s) 2243–2268

    Abstract: Thrombocytopenia results from a variety of conditions, including radiation, chemotherapy, autoimmune disease, bone marrow disorders, pathologic conditions associated with surgical procedures, hematopoietic stem cell transplant (HSCT), and hematologic ... ...

    Abstract Thrombocytopenia results from a variety of conditions, including radiation, chemotherapy, autoimmune disease, bone marrow disorders, pathologic conditions associated with surgical procedures, hematopoietic stem cell transplant (HSCT), and hematologic disorders associated with severe aplastic anemia. Immune thrombocytopenia (ITP) is caused by immune reactions that accelerate destruction and reduce production of platelets. Thrombopoietin (TPO) is a critical component of platelet production pathways, and TPO receptor agonists (TPO-RAs) are important for the management of ITP by increasing platelet production and reducing the need for other treatments. Romiplostim is a TPO-RA approved for use in patients with ITP in the United States, European Union, Australia, and several countries in Africa and Asia, as well as for use in patients with refractory aplastic anemia in Japan and Korea. Romiplostim binds to and activates the TPO receptor on megakaryocyte precursors, thus promoting cell proliferation and viability, resulting in increased platelet production. Through this mechanism, romiplostim reduces the need for other treatments and decreases bleeding events in patients with thrombocytopenia. In addition to its efficacy in ITP, studies have shown that romiplostim is effective in improving platelet counts in various settings, thereby highlighting the versatility of romiplostim. The efficacy of romiplostim in such disorders is currently under investigation. Here, we review the structure, mechanism, pharmacokinetics, and pharmacodynamics of romiplostim. We also summarize the clinical evidence supporting its use in ITP and other disorders that involve thrombocytopenia, including chemotherapy-induced thrombocytopenia, aplastic anemia, acute radiation syndrome, perisurgical thrombocytopenia, post-HSCT thrombocytopenia, and liver disease.
    MeSH term(s) Clinical Trials as Topic ; Humans ; Purpura, Thrombocytopenic, Idiopathic/drug therapy ; Purpura, Thrombocytopenic, Idiopathic/immunology ; Receptors, Fc/chemistry ; Recombinant Fusion Proteins/chemistry ; Recombinant Fusion Proteins/pharmacokinetics ; Thrombopoietin/chemistry ; Thrombopoietin/pharmacokinetics
    Chemical Substances Receptors, Fc ; Recombinant Fusion Proteins ; Thrombopoietin (9014-42-0) ; romiplostim (GN5XU2DXKV)
    Language English
    Publishing date 2021-05-26
    Publishing country New Zealand
    Document type Journal Article ; Review
    ZDB-ID 2451346-5
    ISSN 1177-8881 ; 1177-8881
    ISSN (online) 1177-8881
    ISSN 1177-8881
    DOI 10.2147/DDDT.S299591
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  10. Article: Endocrine, auxological and metabolic profile in children and adolescents with Down syndrome: from infancy to the first steps into adult life.

    Molinari, Silvia / Fossati, Chiara / Nicolosi, Maria Laura / Di Marco, Santo / Faraguna, Martha Caterina / Limido, Francesca / Ocello, Laura / Pellegrinelli, Claudia / Lattuada, Martina / Gazzarri, Alessandra / Lazzerotti, Alessandra / Sala, Debora / Vimercati, Chiara / Capitoli, Giulia / Daolio, Cecilia / Biondi, Andrea / Balduzzi, Adriana / Cattoni, Alessandro

    Frontiers in endocrinology

    2024  Volume 15, Page(s) 1348397

    Abstract: Down syndrome (DS) is the most common chromosomal disorder worldwide. Along with intellectual disability, endocrine disorders represent a remarkable share of the morbidities experienced by children, adolescents and young adults with DS. Auxological ... ...

    Abstract Down syndrome (DS) is the most common chromosomal disorder worldwide. Along with intellectual disability, endocrine disorders represent a remarkable share of the morbidities experienced by children, adolescents and young adults with DS. Auxological parameters are plotted on syndrome-specific charts, as growth rates are reduced compared to healthy age- and gender-matched peers. Furthermore, children with DS are at increased risk for thyroid dysfunctions, diabetes mellitus, osteopenia and obesity compared to general population. Additionally, male individuals with DS often show infertility, while women tend to experience menopause at an overall younger age than healthy controls. Given the recent outstanding improvements in the care of severe DS-related comorbidities, infant mortality has dramatically decreased, with a current average life expectancy exceeding 60 years. Accordingly, the awareness of the specificities of DS in this field is pivotal to timely detect endocrine dysfunctions and to undertake a prompt dedicated treatment. Notably, best practices for the screening and monitoring of pediatric endocrine disorders in DS are still controversial. In addition, specific guidelines for the management of metabolic issues along the challenging period of transitioning from pediatric to adult health care are lacking. By performing a review of published literature, we highlighted the issues specifically involving children and adolescent with DS, aiming at providing clinicians with a detailed up-to-date overview of the endocrine, metabolic and auxological disorders in this selected population, with an additional focus on the management of patients in the critical phase of the transitioning from childhood to adult care.
    MeSH term(s) Humans ; Down Syndrome/metabolism ; Down Syndrome/epidemiology ; Down Syndrome/complications ; Adolescent ; Child ; Endocrine System Diseases/epidemiology ; Endocrine System Diseases/metabolism ; Infant ; Adult ; Male ; Metabolome ; Female ; Child, Preschool
    Language English
    Publishing date 2024-04-08
    Publishing country Switzerland
    Document type Journal Article ; Review ; Research Support, Non-U.S. Gov't
    ZDB-ID 2592084-4
    ISSN 1664-2392
    ISSN 1664-2392
    DOI 10.3389/fendo.2024.1348397
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