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  1. Article: Consensus Statement on Animals' Relationship with Pediatric Oncohematological Patients, on Behalf of Infectious Diseases and Nurse Working Groups of the Italian Association of Pediatric Hematology-Oncology.

    Fiumana, Giulia / Botta, Debora / Dalla Porta, Maria Francesca / Macchi, Simone / Soncini, Elena / Santaniello, Antonio / Paciello, Orlando / Amicucci, Matteo / Cellini, Monica / Cesaro, Simone

    Journal of clinical medicine

    2023  Volume 12, Issue 7

    Abstract: Contact with animals in pediatric oncohematologic patients is associated with many benefits, but the risk of contracting zoonoses, even if low, must be considered by clinicians. In order to assess the awareness about this topic, we surveyed the Italian ... ...

    Abstract Contact with animals in pediatric oncohematologic patients is associated with many benefits, but the risk of contracting zoonoses, even if low, must be considered by clinicians. In order to assess the awareness about this topic, we surveyed the Italian pediatric oncohematology centers, which resulted in heterogeneous responses. The Infectious Diseases Working Group and the Nurse Working Group of the Italian Association of Pediatric Hematology-Oncology, together with veterinarians from the National Federation of Italian Veterinarians, drew up a consensus document to unify the indications to be given to families with the aim of guaranteeing a safe interaction between patients and animals and improving the collaboration of clinicians with veterinarians and families.
    Language English
    Publishing date 2023-03-24
    Publishing country Switzerland
    Document type Journal Article
    ZDB-ID 2662592-1
    ISSN 2077-0383
    ISSN 2077-0383
    DOI 10.3390/jcm12072481
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Allogeneic HSCT for Symptomatic Female X-linked Chronic Granulomatous Disease Carriers.

    Tsilifis, Christo / Torppa, Tuulia / Williams, Eleri J / Albert, Michael H / Hauck, Fabian / Soncini, Elena / Kang, Elizabeth / Malech, Harry / Schuetz, Catharina / von Bernuth, Horst / Slatter, Mary A / Gennery, Andrew R

    Journal of clinical immunology

    2023  Volume 43, Issue 8, Page(s) 1964–1973

    Abstract: X-linked chronic granulomatous disease (XL-CGD) is an inherited disorder of superoxide production, causing failure to generate the oxidative burst in phagocytes. It is characterized by invasive bacterial and fungal infections, inflammation, and chronic ... ...

    Abstract X-linked chronic granulomatous disease (XL-CGD) is an inherited disorder of superoxide production, causing failure to generate the oxidative burst in phagocytes. It is characterized by invasive bacterial and fungal infections, inflammation, and chronic autoimmune disease. While XL-CGD carriers were previously assumed to be healthy, a range of clinical manifestations with significant morbidity have recently been described in a subgroup of carriers with impaired neutrophil oxidative burst due to skewed lyonization. Allogeneic hematopoietic stem cell transplantation (HSCT) is the standard curative treatment for CGD but has rarely been reported in individual symptomatic carriers to date. We undertook a retrospective international survey of outcome of HSCT for symptomatic XL-CGD carriers. Seven symptomatic female XL-CGD carriers aged 1-56 years underwent HSCT in four centers, indicated for severe and recurrent infection, colitis, and autoimmunity. Two patients died from transplant-related complications, following donor engraftment and restoration of oxidative burst. All surviving patients demonstrated resolution of their neutrophil oxidative burst defect with concordant reduction in infection and inflammatory symptoms and freedom from further immunosuppressive therapy. In conclusion, allogeneic HSCT may cure the phagocyte defect in symptomatic XL-CGD carriers and improve their recurrent and disabling infective and inflammatory symptoms but risks transplant-related complications.
    MeSH term(s) Humans ; Female ; Granulomatous Disease, Chronic/diagnosis ; Granulomatous Disease, Chronic/genetics ; Granulomatous Disease, Chronic/therapy ; Retrospective Studies ; Respiratory Burst ; Hematopoietic Stem Cell Transplantation ; Neutrophils
    Language English
    Publishing date 2023-08-24
    Publishing country Netherlands
    Document type Journal Article ; Research Support, N.I.H., Intramural ; Research Support, Non-U.S. Gov't
    ZDB-ID 779361-3
    ISSN 1573-2592 ; 0271-9142
    ISSN (online) 1573-2592
    ISSN 0271-9142
    DOI 10.1007/s10875-023-01570-z
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article ; Online: Letermovir for Cytomegalovirus infection in pediatric patients undergoing allogenic hematopoietic stem cell transplantation: a real-life study by the Infectious Diseases Working Group of Italian Association of Pediatric Hematology-Oncology (AIEOP).

    Galaverna, Federica / Baccelli, Francesco / Zama, Daniele / Tridello, Gloria / Masetti, Riccardo / Soncini, Elena / Mura, Rossella / Barzaghi, Federica / Colombini, Antonella / Prunotto, Giulia / D'Amico, Maria Rosaria / Calore, Elisabetta / Biffi, Alessandra / Perruccio, Katia / Gasperini, Pietro / Oltolini, Chiara / Quagliarella, Francesco / Giacomazzi, Alice / Pagliara, Daria /
    Locatelli, Franco / Cesaro, Simone

    Bone marrow transplantation

    2024  Volume 59, Issue 4, Page(s) 505–512

    Abstract: Letermovir prophylaxis revolutionized the approach to Cytomegalovirus infection in adult hematopoietic stem cell transplant (HCT), while data in pediatric setting are still lacking. We retrospectively analyzed 87 HCT children transplanted in 11 AIEOP ... ...

    Abstract Letermovir prophylaxis revolutionized the approach to Cytomegalovirus infection in adult hematopoietic stem cell transplant (HCT), while data in pediatric setting are still lacking. We retrospectively analyzed 87 HCT children transplanted in 11 AIEOP centers receiving letermovir as off-label indication between January 2020 and November 2022. Letermovir was used as primary, secondary prophylaxis or CMV treatment in 39, 26 and 22 cases, respectively; no discontinuation due to toxicity was reported. Median duration was 100 days (14-256) for primary and 96 days (8-271) for secondary prophylaxis, respectively. None of the patients experienced CMV-clinically significant reactivation during Letermovir primary prophylaxis; one patient developed breakthrough infection during secondary prophylaxis, and 10 and 1 patient experienced asymptomatic CMV-reactivation and CMV-primary infection after drug discontinuation, respectively. Median duration of letermovir in CMV treatment was 40 days (7-134), with 4/22 patients suffering CMV-pneumonia, with an overall response rate of 86.4%. With a median follow-up of 10.7 months (8.2-11.8), estimated 1-year overall survival was 86%; no CMV-related deaths were reported in prophylaxis groups. This is the largest report on Letermovir use in pediatric HCT; real-life data confirm an excellent toxicity profile, with high efficacy as CMV prophylaxis; results in CMV-infection treatment should be investigated in larger, prospective trials.
    MeSH term(s) Adult ; Humans ; Child ; Cytomegalovirus ; Retrospective Studies ; Prospective Studies ; Antiviral Agents/adverse effects ; Cytomegalovirus Infections/drug therapy ; Cytomegalovirus Infections/etiology ; Cytomegalovirus Infections/prevention & control ; Communicable Diseases ; Hematopoietic Stem Cell Transplantation/adverse effects ; Hematology ; Italy ; Acetates ; Quinazolines
    Chemical Substances letermovir (1H09Y5WO1F) ; Antiviral Agents ; Acetates ; Quinazolines
    Language English
    Publishing date 2024-01-25
    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/s41409-024-02209-2
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article: Primary Immunodeficiencies and Oncological Risk: The Experience of the Children's Hospital of Brescia.

    Maffeis, Marianna / Notarangelo, Lucia Dora / Schumacher, Richard Fabian / Soncini, Elena / Soresina, Annarosa / Lanfranchi, Arnalda / Porta, Fulvio

    Frontiers in pediatrics

    2019  Volume 7, Page(s) 232

    Abstract: Background and aims: ...

    Abstract Background and aims:
    Language English
    Publishing date 2019-06-19
    Publishing country Switzerland
    Document type Journal Article
    ZDB-ID 2711999-3
    ISSN 2296-2360
    ISSN 2296-2360
    DOI 10.3389/fped.2019.00232
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: Long-Term Survival After Hematopoietic Stem Cell Transplantation for Complete STAT1 Deficiency.

    Naviglio, Samuele / Soncini, Elena / Vairo, Donatella / Lanfranchi, Arnalda / Badolato, Raffaele / Porta, Fulvio

    Journal of clinical immunology

    2017  Volume 37, Issue 7, Page(s) 701–706

    Abstract: Purpose: Complete signal transducer and activator of transcription 1 (STAT1) deficiency is a rare autosomal recessive condition characterized by impairment of intracellular signaling from both type I and type II interferons (IFN). Affected patients are ... ...

    Abstract Purpose: Complete signal transducer and activator of transcription 1 (STAT1) deficiency is a rare autosomal recessive condition characterized by impairment of intracellular signaling from both type I and type II interferons (IFN). Affected patients are prone to early severe mycobacterial and viral infections, which usually result in death before 18 months of age. We previously reported a patient affected by complete STAT1 deficiency who underwent hematopoietic stem cell transplantation (HSCT). Here, we describe the transplantation procedures and long-term outcomes.
    Methods: The patient, who had suffered multiple life-threatening mycobacterial and viral infections in the first years of life, underwent HSCT at 4 years of age from a partially matched (HLA compatibility 8/10) unrelated donor after a myeloablative conditioning regimen consisting of busulfan, cyclophosphamide, and anti-thymocyte globulin.
    Results: Hematological reconstitution was detected at d+15, with full donor engraftment demonstrated by molecular analysis of leukocytes. Several complications occurred in the post-transplantation phase, including acute graft versus host disease, posterior reversible encephalopathy, thrombotic thrombocytopenic purpura, bilateral keratoconjunctivitis with complete loss of vision, and chronic lower limb lymphedema. Analysis of STAT1 in CD3
    Conclusions: Notably, no severe infections occurred after discharge (day + 90) during a 9-year follow-up, suggesting that normal response to IFNs in hematopoietic cells is sufficient to provide protection in humans.
    MeSH term(s) Child, Preschool ; Hematopoietic Stem Cell Transplantation ; Humans ; Immunologic Deficiency Syndromes/therapy ; Male ; STAT1 Transcription Factor/deficiency ; Treatment Outcome
    Chemical Substances STAT1 Transcription Factor ; STAT1 protein, human
    Language English
    Publishing date 2017-08-16
    Publishing country Netherlands
    Document type Case Reports ; Journal Article
    ZDB-ID 779361-3
    ISSN 1573-2592 ; 0271-9142
    ISSN (online) 1573-2592
    ISSN 0271-9142
    DOI 10.1007/s10875-017-0430-6
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  6. Article ; Online: CD34+ Stem Cell Selection and CD3+ T Cell Add-Back from Matched Unrelated Adult Donors in Children with Primary Immunodeficiencies and Hematological Diseases.

    Porta, Fulvio / Comini, Marta / Soncini, Elena / Carracchia, Giulia / Maffeis, Marianna / Pintabona, Vincenzo / Bolda, Federica / Beghin, Alessandra / Schumacher, Richard Fabian / Lanfranchi, Arnalda

    Transplantation and cellular therapy

    2021  Volume 27, Issue 5, Page(s) 426.e1–426.e9

    Abstract: Less than 25% of children who require hematopoietic stem cell transplantation (HSCT) for primary immunodeficiencies (PIDs) or genetic hematological diseases have an HLA-identical sibling. For them, a matched unrelated donor (MUD), although baring a ... ...

    Abstract Less than 25% of children who require hematopoietic stem cell transplantation (HSCT) for primary immunodeficiencies (PIDs) or genetic hematological diseases have an HLA-identical sibling. For them, a matched unrelated donor (MUD), although baring a greater risk of graft failure, delayed engraftment and immune reconstitution, and severe graft-versus-host disease (GvHD), represents a valid alternative. The stem cell source is also important, as unprocessed peripheral blood stem cells (PBSCs) contain 5 to 10 times more T cells than bone marrow (BM)-derived grafts, a major risk especially for small children with PID. A CD34+ positive selection can mitigate HLA compatibility issues, but the resulting CD3+ T cell depletion hampers engraftment and facilitates infections. To mitigate those problems, we decided to add back a certain number of T cells (30 × 10
    MeSH term(s) Adult ; Child ; Child, Preschool ; Graft vs Host Disease ; Hematologic Diseases ; Hematopoietic Stem Cell Transplantation ; Humans ; Infant ; Retrospective Studies
    Language English
    Publishing date 2021-01-24
    Document type Journal Article
    ZDB-ID 3062231-1
    ISSN 2666-6367
    ISSN (online) 2666-6367
    DOI 10.1016/j.jtct.2021.01.020
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  7. Article ; Online: Clinical and transcriptomic characteristics of a novel SMARCD2 mutation that disrupts neutrophil maturation and function.

    Dotta, Laura / Baresi, Giulia / Tamassia, Nicola / Calzetti, Federica / Bianchetto-Aguilera, Francisco / Gasperini, Sara / Gardiman, Elisa / Chiarini, Marco / Moratto, Daniele / Martellosio, Giovanni / Serana, Federico / Micheletti, Moira / Tregambe, Daniela / Pintabona, Vincenzo / Soncini, Elena / Meini, Antonella / Girelli, Maria Federica / Beghin, Alessandra / Lanfranchi, Arnalda /
    Bugatti, Mattia / Brugnoni, Duilio / Soresina, Annarosa / Plebani, Alessandro / Cassatella, Marco / Vermi, William / Porta, Fulvio / Badolato, Raffaele

    Pediatric blood & cancer

    2023  Volume 70, Issue 12, Page(s) e30671

    Abstract: We report a novel case of SMARCD2 (SWI/SNF-related, matrix-associated, actin-dependent regulator of chromatin, subfamily D, member 2) mutation successfully treated with hematopoietic stem cell transplantation. The female patient presented delayed cord ... ...

    Abstract We report a novel case of SMARCD2 (SWI/SNF-related, matrix-associated, actin-dependent regulator of chromatin, subfamily D, member 2) mutation successfully treated with hematopoietic stem cell transplantation. The female patient presented delayed cord separation, chronic diarrhea, skin abscesses, skeletal dysmorphisms, and neutropenia with specific granule deficiency. Analysis of the transcriptomic profile of peripheral blood sorted mature and immature SMARCD2 neutrophils showed defective maturation process that associated with altered expression of genes related to specific, azurophilic, and gelatinase granules, such as LTF, CRISP3, PTX3, and CHI3L1. These abnormalities account for the prevalence of immature neutrophils in the peripheral blood, impaired function, and deregulated inflammatory responses.
    Language English
    Publishing date 2023-09-15
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2131448-2
    ISSN 1545-5017 ; 1545-5009
    ISSN (online) 1545-5017
    ISSN 1545-5009
    DOI 10.1002/pbc.30671
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  8. Article ; Online: Prophylaxis With Trimethoprim/Sulfamethoxazole Is Not Necessary in Children With Solid Tumors Treated With Low-medium Intensity Chemotherapy.

    Meazza, Cristina / Galaverna, Federica / Petris, Maria Grazia / Zama, Daniele / La Spina, Milena / Muggeo, Paola / Ziino, Ottavio / Cellini, Monica / Soncini, Elena / Barone, Angelica / De Santis, Raffaella / Perruccio, Katia / Mura, Rossella / Pascale, Silvia / Mercolini, Federico / Cesaro, Simone

    The Pediatric infectious disease journal

    2021  Volume 40, Issue 4, Page(s) 354–355

    Abstract: Prophylaxis of Pneumocystis jiroveci pneumonia (PJP) with trimethoprim/sulfamethoxazole is a standard of care for children with hematologic malignancies, while its use in solid tumor patients is still debated. A retrospective study focusing on the use of ...

    Abstract Prophylaxis of Pneumocystis jiroveci pneumonia (PJP) with trimethoprim/sulfamethoxazole is a standard of care for children with hematologic malignancies, while its use in solid tumor patients is still debated. A retrospective study focusing on the use of PJP prophylaxis in patients with solid tumors was performed among 16 AIEOP centers: 1046/2863 patients did not receive prophylaxis and no cases of PJP were reported.
    MeSH term(s) Adolescent ; Anti-Bacterial Agents/administration & dosage ; Anti-Bacterial Agents/standards ; Antibiotic Prophylaxis/standards ; Antibiotic Prophylaxis/statistics & numerical data ; Antineoplastic Agents/administration & dosage ; Antineoplastic Agents/therapeutic use ; Child ; Child, Preschool ; Humans ; Immunocompromised Host ; Neoplasms/drug therapy ; Pneumonia, Pneumocystis/microbiology ; Pneumonia, Pneumocystis/prevention & control ; Retrospective Studies ; Trimethoprim, Sulfamethoxazole Drug Combination/administration & dosage ; Trimethoprim, Sulfamethoxazole Drug Combination/standards
    Chemical Substances Anti-Bacterial Agents ; Antineoplastic Agents ; Trimethoprim, Sulfamethoxazole Drug Combination (8064-90-2)
    Language English
    Publishing date 2021-04-06
    Publishing country United States
    Document type Journal Article ; Multicenter Study
    ZDB-ID 392481-6
    ISSN 1532-0987 ; 0891-3668
    ISSN (online) 1532-0987
    ISSN 0891-3668
    DOI 10.1097/INF.0000000000003044
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Article ; Online: Safety and efficacy of brincidofovir for Adenovirus infection in children receiving allogeneic stem cell transplantation: an AIEOP retrospective analyses.

    Perruccio, Katia / Menconi, Mariacristina / Galaverna, Federica / Pagliara, Daria / Carraro, Francesca / Fagioli, Franca / Calore, Elisabetta / Biffi, Alessandra / Baretta, Valentina / Massei, Maria Speranza / Capolsini, Ilaria / Faraci, Maura / Verna, Marta / Soncini, Elena / Caniglia, Maurizio / Locatelli, Franco / Cesaro, Simone

    Bone marrow transplantation

    2021  Volume 56, Issue 12, Page(s) 3104–3107

    MeSH term(s) Adenoviridae Infections/drug therapy ; Antiviral Agents/therapeutic use ; Cytosine/analogs & derivatives ; Cytosine/pharmacology ; Cytosine/therapeutic use ; Hematopoietic Stem Cell Transplantation ; Humans ; Organophosphonates ; Retrospective Studies
    Chemical Substances Antiviral Agents ; Organophosphonates ; brincidofovir (6794O900AX) ; Cytosine (8J337D1HZY)
    Language English
    Publishing date 2021-10-04
    Publishing country England
    Document type Letter
    ZDB-ID 632854-4
    ISSN 1476-5365 ; 0268-3369 ; 0951-3078
    ISSN (online) 1476-5365
    ISSN 0268-3369 ; 0951-3078
    DOI 10.1038/s41409-021-01285-y
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  10. Article ; Online: Antibiotic prophylaxis and management of infections in pediatric hematopoietic stem cell transplantation: a survey from the Stem Cell Transplant and the Infectious Disease Working Groups of the AIEOP network.

    Zama, Daniele / Masetti, Riccardo / Baccelli, Francesco / Leardini, Davide / Muratore, Edoardo / Abram, Nicoletta / Vendemini, Francesca / Biffi, Alessandra / Perruccio, Katia / D'Amico, Maria Rosaria / Faraci, Maura / Tintori, Veronica / Spirito, Anita / Lo Nigro, Luca / Locatelli, Franco / Luksch, Roberto / Saglio, Francesco / Santoro, Nicola / Soncini, Elena /
    Zecca, Marco / Ziino, Ottavio / Prete, Arcangelo / Pagliara, Daria / Cesaro, Simone

    Bone marrow transplantation

    2022  Volume 57, Issue 12, Page(s) 1851–1853

    MeSH term(s) Child ; Humans ; Antibiotic Prophylaxis ; Hematopoietic Stem Cell Transplantation ; Communicable Diseases ; Stem Cell Transplantation
    Language English
    Publishing date 2022-10-07
    Publishing country England
    Document type Letter
    ZDB-ID 632854-4
    ISSN 1476-5365 ; 0268-3369 ; 0951-3078
    ISSN (online) 1476-5365
    ISSN 0268-3369 ; 0951-3078
    DOI 10.1038/s41409-022-01793-5
    Database MEDical Literature Analysis and Retrieval System OnLINE

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