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  • 1
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2015-02, Vol.50 (2), p.181-188
    Description: We analyzed the outcome of 243 children with high-risk (HR) AML in first CR1 enrolled in the AIEOP-2002/01 protocol, who were given either allogeneic (ALLO; n=141) or autologous (AUTO; n=102) hematopoietic SCT (HSCT), depending on the availability of a HLA-compatible sibling. Infants, patients with AML-M7, or complex karyotype or those with FLT3-ITD, were eligible to be transplanted also from alternative donors. All patients received a myeloablative regimen combining busulfan, cyclophosphamide and melphalan; [corrected] AUTO-HSCT patients received BM cells in most cases, while in children given ALLO-HSCT stem cell source was BM in 96, peripheral blood in 19 and cord blood in 26. With a median follow-up of 57 months (range 12-130), the probability of disease-free survival (DFS) was 73% and 63% in patients given either ALLO- or AUTO-HSCT, respectively (P=NS). Although the cumulative incidence (CI) of relapse was lower in ALLO- than in AUTO-HSCT recipients (17% vs 28%, respectively; P=0.043), the CI of TRM was 7% in both groups. Patients transplanted with unrelated donor cord blood had a remarkable 92.3% 8-year DFS probability. Altogether, these data confirm that HSCT is a suitable option for preventing leukemia recurrence in HR children with CR1 AML.
    Subject(s): Autografts ; Follow-Up Studies ; Leukemia, Myeloid, Acute - pathology ; Humans ; Child, Preschool ; Hematopoietic Stem Cell Transplantation ; Infant ; Male ; Survival Rate ; Abnormal Karyotype ; Cord Blood Stem Cell Transplantation ; Leukemia, Myeloid, Acute - mortality ; fms-Like Tyrosine Kinase 3 - genetics ; Disease-Free Survival ; Allografts ; Adolescent ; Myeloablative Agonists - administration & dosage ; Female ; Transplantation Conditioning - methods ; Child ; Leukemia, Myeloid, Acute - therapy ; Leukemia, Myeloid, Acute - genetics ; Transplantation ; Health aspects ; Patient outcomes ; Hematopoietic stem cells ; Index Medicus
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 2
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2015-02-01, Vol.50 (2), p.320
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: Alma/SFX Local Collection
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  • 3
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2010-11, Vol.45 (11), p.1663-1664
    Subject(s): Biological and medical sciences ; Medical sciences ; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Bone marrow, stem cells transplantation. Graft versus host reaction ; Transfusions. Complications. Transfusion reactions. Cell and gene therapy ; Neoplasms - surgery ; Humans ; Child, Preschool ; Serotonin Antagonists - therapeutic use ; Infant ; Male ; Nausea - prevention & control ; Nausea - etiology ; Quinuclidines - therapeutic use ; Antiemetics - therapeutic use ; Adolescent ; Bone Marrow Transplantation - adverse effects ; Isoquinolines - therapeutic use ; Vomiting - etiology ; Female ; Retrospective Studies ; Child ; Vomiting - prevention & control ; Prevention ; Complications and side effects ; Usage ; Bone marrow ; Nausea ; Transplantation ; Drug therapy ; Health aspects ; Risk factors ; Palonosetron ; Index Medicus
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 4
    Language: English
    In: British journal of haematology, 2014-06, Vol.165 (5), p.673-681
    Description: Summary Allogeneic haematopoietic stem cell transplantation (HSCT) is the only curative option for patients with Diamond Blackfan anaemia (DBA). We report the transplantation outcome of 30 Italian DBA patients referred to the Italian Association of Paediatric Haematology and Oncology Registry between 1990 and 2012. This is one of the largest national registry cohorts of transplanted DBA patients. Most patients (83%) were allografted after 2000. A matched sibling donor was employed in 16 patients (53%), the remaining 14 patients (47%) were transplanted from matched unrelated donors. Twenty‐eight of the 30 patients engrafted. One patient died at day +6 due to veno‐occlusive disease without achieving neutrophil recovery and another patient remained transfusion‐dependent despite the presence of a full donor chimerism. The 5‐year overall survival and transplant‐related mortality was 74·4% and 25·6%, respectively. Patients younger than 10 years as well as those transplanted after 2000 showed a significantly higher overall survival and a significantly lower risk of transplant‐related mortality. No difference between donor type was observed. Our data suggest that allogeneic HSCT from a related or unrelated donor was a reasonable alternative to transfusion therapy in young and well chelated DBA patients.
    Subject(s): BMT ; iron overload ; neoplasm ; congenital red cell aplasia ; Diamond‐Blackfan anaemia ; Hematologic and hematopoietic diseases ; Anemias. Hemoglobinopathies ; Diseases of red blood cells ; Biological and medical sciences ; Multiple tumors. Solid tumors. Tumors in childhood (general aspects) ; Medical sciences ; Tumors ; Graft vs Host Disease - epidemiology ; Age Factors ; Follow-Up Studies ; Humans ; Risk Factors ; Child, Preschool ; Graft Survival ; Infant ; Male ; Anemia, Diamond-Blackfan - mortality ; Hematopoietic Stem Cell Transplantation - mortality ; Cause of Death ; Adolescent ; Hematopoietic Stem Cell Transplantation - adverse effects ; Survival Analysis ; Anemia, Diamond-Blackfan - therapy ; Female ; Italy - epidemiology ; Registries ; Hematopoietic Stem Cell Transplantation - methods ; Child ; Graft vs Host Disease - etiology ; Pediatrics ; Transplantation ; Mortality ; Hematopoietic stem cells ; Stem cells ; Index Medicus
    ISSN: 0007-1048
    E-ISSN: 1365-2141
    Source: Alma/SFX Local Collection
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 5
    Language: English
    In: Haematologica (Roma), 2013-08-01, Vol.98 (8), p.1273-1281
    Description: Children with high-risk acute lymphoblastic leukemia in first complete remission can benefit from allogeneic hematopoietic stem cell transplantation. We analyzed the outcome of 211 children with high-risk acute lymphoblastic leukemia in first complete remission who were given an allogeneic transplant between 1990 and 2008; the outcome of patients who, despite having an indication for transplantation and a suitable donor, did not receive the allograft for different reasons in the same time period was not analyzed. Sixty-nine patients (33%) were transplanted between 1990 and 1999, 58 (27%) between 2000 and 2005, and 84 (40%) between 2005 and 2008. A matched family donor was employed in 138 patients (65%) and an unrelated donor in 73 (35%). The 10-year probabilities of overall and disease-free survival were 63.4% and 61%, respectively. The 10-year cumulative incidences of transplantation-related mortality and relapse were 15% and 24%, respectively. After 1999, no differences in either disease-free survival or transplant-related mortality were observed in patients transplanted from unrelated or matched family donors. In multivariate analysis, grade IV acute graft-versus-host disease was an independent factor associated with worse disease-free survival. By contrast, grade I acute graft-versus-host disease and age at diagnosis between 1 and 9 years were favorable prognostic variables. Our study, not intended to evaluate whether transplantation is superior to chemotherapy for children with acute lymphoblastic leukemia in first complete remission and high-risk features, shows that the allograft cured more than 60% of these patients; in the most recent period, the outcome of recipients of grafts from matched family and unrelated donors was comparable.
    Subject(s): Graft vs Host Disease - epidemiology ; Follow-Up Studies ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - surgery ; Humans ; Risk Factors ; Child, Preschool ; Infant ; Male ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - mortality ; Treatment Outcome ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - diagnosis ; Hematopoietic Stem Cell Transplantation - mortality ; Remission Induction ; Transplantation, Homologous - mortality ; Disease-Free Survival ; Adolescent ; Hematopoietic Stem Cell Transplantation - adverse effects ; Female ; Italy - epidemiology ; Registries ; Child ; Graft vs Host Disease - diagnosis ; Transplantation, Homologous - adverse effects ; Index Medicus ; Original and Brief Reports
    ISSN: 0390-6078
    E-ISSN: 1592-8721
    Source: HighWire Press (Free Journals)
    Source: PubMed Central
    Source: Directory of Open Access Journals
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 6
    Language: English
    In: Blood, 2011, Vol.118 (25), p.6683-6690
    Description: Acute lymphoblastic leukemia (ALL) in second complete remission is one of the most common indications for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients. We compared the outcome after HCST of adolescents, aged 14 to 18 years, with that of children (ie, patients 〈 14 years of age). Enrolled in the study were 395 patients given the allograft between January 1990 and December 2007; both children (334) and adolescents (61) were transplanted in the same pediatric institutions. All patients received a myeloablative regimen that included total body irradiation in the majority of them. The donor was an HLA-identical sibling for 199 patients and an unrelated volunteer in the remaining 196 patients. Children and adolescents had a comparable cumulative incidence of transplantation-related mortality, disease recurrence, and of both acute and chronic graft-versus-host disease. The 10-year probability of overall survival and event-free survival for the whole cohort of patients were 57% (95% confidence interval, 52%-62%) and 54% (95% confidence interval, 49%-59%), respectively, with no difference between children and adolescents. This study documents that adolescents with ALL in second complete remission given HSCT in pediatric centers have an outcome that does not differ from that of patients younger than 14 years of age.
    Subject(s): Hematologic and hematopoietic diseases ; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis ; Biological and medical sciences ; Medical sciences ; Multivariate Analysis ; Outcome Assessment, Health Care - statistics & numerical data ; Recurrence ; Chemoradiotherapy - methods ; Humans ; Proportional Hazards Models ; Child, Preschool ; Infant ; Male ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - mortality ; Survival Rate ; Remission Induction ; Transplantation, Homologous ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy ; Time Factors ; Adolescent ; Hematopoietic Stem Cell Transplantation - adverse effects ; Survival Analysis ; Female ; Hematopoietic Stem Cell Transplantation - methods ; Child ; Graft vs Host Disease - etiology ; Infant, Newborn ; Cohort Studies ; Index Medicus ; Abridged Index Medicus
    ISSN: 0006-4971
    E-ISSN: 1528-0020
    Source: HighWire Press (Free Journals)
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
    Source: American Society of Hematology
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 7
    Language: English
    In: Haematologica (Roma), 2008-11-01, Vol.93 (11), p.1694-1701
    Description: 1 Oncoematologia Pediatrica, Dipartimento di Pediatria, Università di Padova, Padova 2 Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo, Università di Pavia, Pavia 3 Dipartimento di Ematologia e Oncologia Pediatrica, IRCCS Giannina Gaslini, Genova 4 Clinica Pediatrica , Università di Brescia, Spedali Civili di Brescia 5 Clinica Pediatrica, Università Milano-Bicocca, Ospedale Nuovo S. Gerardo, Monza 6 Oncologia ed Ematologia Pediatrica "Lalla Seràgnoli", Ospedale S. Orsola Malpighi, Università di Bologna 7 Oncoematologia Pediatrica, Ospedale Pausilipon, Napoli 8 Centro Trapianti Trapianti di Midollo, IRCCS Burlo Garofolo, Trieste 9 Dipartimento di Oncoematologia Pediatrica, Azienda Ospedaliero-Universitaria Meyer, Firenze and 10 Oncoematologia Pediatrica, Ospedale Infantile Regina Margherita, Torino, Italy Correspondence: Simone Cesaro, MD, Oncoematologia Pediatrica, Dipartimento di Pediatria, Università di Padova, Via Giustiniani 3,35128, Padova Italy. E-mail: simone.cesaro{at}unipd.it Background: Hemophagocytic lymphohistiocytosis is a life-threatening disease. Hematopoietic stem cell transplantation still represents the treatment of choice for most patients with this disease. Design and Methods: We retrospectively analyzed 61 patients with hemophagocytic lymphohistiocytosis who underwent HSCT over a 17-year period at nine centers affiliated to the Italian Pediatric Hematology Oncology Association (AIEOP). The median time from diagnosis to hematopoietic stem cell transplantation was 0.6 years (range, 0.13–5). The donor for the first hematopoietic stem cell transplantation was either a relative (43%) or an unrelated volunteer (57%). Fifty-four patients (89%) had a complete genetic study, which led to the diagnoses of FHL2, due to perforin defect (21 patients), FHL3, due to Munc 13-4 defect (14 patients), Griscelli disease (2 patients), X-linked lymphoproliferative disease (1 patient), and CATCH22 syndrome (1 patient). No mutations were found in the remaining 15 patients. Twenty-one patients had neurological involvement at diagnosis. Results: Three patients failed to engraft. Grade II–IV acute and chronic graft-versus-host disease occurred in 31% and 17% of patients, respectively. Overall, 39 patients are alive (64%), 15 died of toxicity, 6 of progressive disease and 1 of sudden death. The 8-year overall survival probability was 58.6% (95% confidence interval, 42–72), while the cumulative incidence of transplantation-related mortality was 25.7% (95% confidence interval, 16–40). The outcome of patients with a known genetic defect was comparable to that of patients without mutation. Neurological sequelae were reported in seven patients, six of whom had central nervous system disease at diagnosis. Conclusions: These data confirm that hematopoietic stem cell transplantation represents a curative treatment for a large proportion of patients with hemophagocytic lymphohistiocytosis, irrespective of the underlying genetic defect. Key words: hemophagocytic lymphohistiocytosis, perforin defect, hematopoietic stem cell transplantation, transplantation-related mortality, children. Related Article Hematopoietic stem cell transplantation: 40 years of continuous progress and evolution Fulvio Porta, Franco Locatelli, Giuseppe Roberto Burgio Haematologica 2008 93: 1607-1610. [Full Text] [PDF]
    Subject(s): Hematologic and hematopoietic diseases ; Biological and medical sciences ; Medical sciences ; Graft vs Host Disease - epidemiology ; Hematologic Neoplasms - mortality ; Humans ; Survivors ; Child, Preschool ; Infant ; Antineoplastic Agents - therapeutic use ; Combined Modality Therapy ; Hematologic Neoplasms - surgery ; Antineoplastic Combined Chemotherapy Protocols - therapeutic use ; Hematopoietic Stem Cell Transplantation - adverse effects ; Survival Analysis ; Hematologic Neoplasms - drug therapy ; Lymphohistiocytosis, Hemophagocytic - surgery ; Italy ; Retrospective Studies ; Hematopoietic Stem Cell Transplantation - methods ; Child ; Infant, Newborn
    ISSN: 0390-6078
    E-ISSN: 1592-8721
    Source: HighWire Press (Free Journals)
    Source: Directory of Open Access Journals
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  • 8
    Language: English
    In: Haematologica (Roma), 2016-03, Vol.101 (3), p.319-327
    Description: The natural history of Fanconi anemia remains hard to establish because of its rarity and its heterogeneous clinical presentation; since 1994, the Italian Fanconi Anemia Registry has collected clinical, epidemiological and genetic data of Italian Fanconi Anemia patients. This registry includes 180 patients with a confirmed diagnosis of Fanconi anemia who have either been enrolled prospectively, at diagnosis, or later on. After enrollment, follow-up data were periodically collected to assess the clinical course, possible complications and long-term survival; the median follow up was 15.6 years. The main goal of the study was to describe the natural history of Fanconi anemia, focusing on the following variables: family history, disease presentation, development of hematological manifestations, development of malignancies, occurrence of hematopoietic stem cell transplantation and survival. Typical morphological and/or hematological abnormalities and/or growth retardation were the most common manifestations at diagnosis; the majority of patients (77%) exhibited hematological abnormalities at the initial presentation, and almost all (96%) eventually developed hematological manifestations. More than half of the patients (57%) underwent a bone-marrow transplant. The occurrence of cancer was quite rare at diagnosis, whereas the cumulative incidence of malignancies at 10, 20 and 30 years was 5%, 8% and 22%, respectively, for hematological cancers and 1%, 15% and 32%, respectively, for solid tumors. Overall survival at 10, 20 and 30 years were 88%, 56% and 37%, respectively; the main causes of death were cancer, complications of the hematological presentation and complications of transplantation. These data clearly confirm the detrimental outcome of Fanconi anemia, with no major improvement in the past decades.
    Subject(s): Graft vs Host Disease - therapy ; Hematologic Neoplasms - therapy ; Hematologic Neoplasms - mortality ; Humans ; Middle Aged ; Child, Preschool ; Infant ; Male ; Opportunistic Infections - mortality ; Hematologic Neoplasms - pathology ; Opportunistic Infections - therapy ; Fanconi Anemia - mortality ; Graft vs Host Disease - mortality ; Bone Marrow Transplantation ; Adult ; Fanconi Anemia - pathology ; Female ; Registries ; Hematologic Neoplasms - complications ; Child ; Infant, Newborn ; Opportunistic Infections - complications ; Fanconi Anemia - complications ; Disease Progression ; Graft vs Host Disease - pathology ; Bone Marrow - pathology ; Adolescent ; Survival Analysis ; Italy ; Fanconi Anemia - therapy ; Longitudinal Studies ; Opportunistic Infections - pathology ; Index Medicus
    ISSN: 0390-6078
    E-ISSN: 1592-8721
    Source: HighWire Press (Free Journals)
    Source: PubMed Central
    Source: Directory of Open Access Journals
    Source: © ProQuest LLC All rights reserved〈img src="https://exlibris-pub.s3.amazonaws.com/PQ_Logo.jpg" style="vertical-align:middle;margin-left:7px"〉
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  • 9
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2015-02-04, Vol.50 (2), p.320-320
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
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  • 10
    Language: English
    In: Bone marrow transplantation (Basingstoke), 2009-03-01, Vol.43 (S1), p.S375
    Description: One of the major side effects poorly tolerated, especially in children, is represented by emesis post-chemotherapy. The use of antiemetic during chemotherapy (three to four doses for day) is necessary to reduce this complication. In this work was evaluated using a single dose of palonosetron intravenous for the prevention of nausea and vomiting secondary to chemotherapies.
    Subject(s): Complications and side effects ; Chemotherapy ; Bone marrow ; Dosage and administration ; Transplantation ; Children ; Research ; Health aspects ; Palonosetron ; Cancer
    ISSN: 0268-3369
    E-ISSN: 1476-5365
    Source: Nature Open Access
    Source: Academic Search Ultimate
    Source: Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
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