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Article: Status quo of allogeneic stem cell transplantation for patients with sickle cell disease using matched unrelated donors.

Oevermann, L / Sodani, Pietro

Hematology/oncology and stem cell therapy

2020 Volume 13, Issue 2, Page(s) 116–119

Abstract: This mini review is based on an oral presentation reflecting the current status quo of allogeneic hematopoietic stem cell transplantation (HSCT) for patients with sickle cell disease (SCD) using matched unrelated donors (MUDs) presented at the EBMT ... ...

Abstract	This mini review is based on an oral presentation reflecting the current status quo of allogeneic hematopoietic stem cell transplantation (HSCT) for patients with sickle cell disease (SCD) using matched unrelated donors (MUDs) presented at the EBMT Sickle Disease Meeting held in Regensburg, Germany, in May 2019. Although the clinical trial landscape for MUD HSCT in patients with SCD is limited to date, some attempts to improve patient outcome in terms of overall survival and event-free survival have been made recently, including optimization of conditioning regimens and prevention of engraftment failure as well as graft-versus-host disease. The results achieved by these approaches are summarized in this review and are still unsatisfactory. Whether new haploidentical transplantation protocols will achieve superior results and are able to replace MUD HSCT for patients with SCD remains to be elucidated.
MeSH term(s)	Anemia, Sickle Cell/therapy ; Female ; Hematopoietic Stem Cell Transplantation/methods ; Humans ; Male ; Transplantation Conditioning/methods ; Transplantation, Homologous/methods ; Unrelated Donors
Language	English
Publishing date	2020-03-12
Publishing country	England
Document type	Journal Article ; Review
ZDB-ID	2651893-4
ISSN	1658-3876
ISSN	1658-3876
DOI	10.1016/j.hemonc.2019.12.004
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Article ; Online: HLA-haploidentical hematopoietic stem cell transplantation in pediatric patients with hemoglobinopathies: current practice and new approaches.

Oevermann, Lena / Schulte, Johannes H / Hundsdörfer, Patrick / Hakimeh, Dani / Kogel, Friederike / Lang, Peter / Corbacioglu, Selim / Eggert, Angelika / Sodani, Pietro

Bone marrow transplantation

2019 Volume 54, Issue Suppl 2, Page(s) 743–748

Abstract: We review current approaches in HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for pediatric patients with hemoglobinopathies with a focus on recent developments using TCRα/β+/CD19+ depleted grafts in patients with β-thalassemia ... ...

Abstract	We review current approaches in HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for pediatric patients with hemoglobinopathies with a focus on recent developments using TCRα/β+/CD19+ depleted grafts in patients with β-thalassemia major (TM) or sickle cell disease (SCD) in two European transplant units. Eleven TM and three SCD patients (Roma cohort) received a preparative regimen consisting of busulfan/thiotepa/cyclophosphamide/ATG preceded by fludarabine/hydroxyurea/azathioprine. The preparative regimen for 5 SCD patients included treosulfan/thiotepa/fludarabine/ATG (Berlin pilot cohort). All grafts were PBSC engineered by TCR-α/β+/CD19+ depletion. In both cohorts, rates for graft failure, treatment related mortality (TRM) and GvHD were encouraging. Overall survival (OS) and disease-free survival (DFS) in the Roma cohort were 84 and 69%, respectively, while OS and DFS are 100% in the Berlin cohort. Immune reconstitution was satisfactory. Although asymptomatic viral reactivation was common, no severe viral infection occured. These data confirm that TCR-α/β+/CD19+ depletion is a well-suited haplo-HSCT strategy for children with hemoglobinopathies. We discuss the results in the context of additional optimization strategies and introduce our concepts for multicenter trial protocols in Germany.
MeSH term(s)	Adolescent ; Female ; Humans ; Male ; Young Adult ; Hematopoietic Stem Cell Transplantation/methods ; Hemoglobinopathies ; Pilot Projects ; Transplantation Conditioning/methods ; Transplantation, Haploidentical/methods
Language	English
Publishing date	2019-07-23
Publishing country	England
Document type	Journal Article ; Research Support, Non-U.S. Gov't ; Review
ZDB-ID	632854-4
ISSN	1476-5365 ; 0268-3369 ; 0951-3078
ISSN (online)	1476-5365
ISSN	0268-3369 ; 0951-3078
DOI	10.1038/s41409-019-0598-x
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Article ; Online: One single bone marrow harvesting from donors under 3 years of age: assessing safety and efficacy of the procedure.

Paciaroni, Katia / Alfieri, Cecilia / Isgrò, Antonella / De Angelis, Gioia / Ribersani, Michela / Marziali, Marco / Dauri, Mario / Sodani, Pietro / Gaziev, Javid

Bone marrow transplantation

2018 Volume 54, Issue 7, Page(s) 1121–1123

Abstract: To candidate children as bone marrow donors raises two main concerns: donor safety and adequate marrow cell dose. Data in the field are limited and guidelines for child donor care management are lacking. In this context, we herein report the experience ... ...

Abstract	To candidate children as bone marrow donors raises two main concerns: donor safety and adequate marrow cell dose. Data in the field are limited and guidelines for child donor care management are lacking. In this context, we herein report the experience collected in our center by comparing very-young donors (defined as age ≤ 3 years) with young donors (defined as age > 3 years) who donated bone marrow (BM) for patients affected by beta-globin disorders.
MeSH term(s)	Bone Marrow Transplantation/methods ; Child, Preschool ; Female ; Humans ; Male ; Tissue Donors ; Tissue and Organ Harvesting/methods
Language	English
Publishing date	2018-12-05
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/s41409-018-0415-y
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Article ; Online: Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia.

Lucarelli, Guido / Isgrò, Antonella / Sodani, Pietro / Gaziev, Javid

Cold Spring Harbor perspectives in medicine

2012 Volume 2, Issue 5, Page(s) a011825

Abstract: The globally widespread single-gene disorders β-thalassemia and sickle cell anemia (SCA) can only be cured by allogeneic hematopoietic stem cell transplantation (HSCT). HSCT treatment of thalassemia has substantially improved over the last two decades, ... ...

Abstract	The globally widespread single-gene disorders β-thalassemia and sickle cell anemia (SCA) can only be cured by allogeneic hematopoietic stem cell transplantation (HSCT). HSCT treatment of thalassemia has substantially improved over the last two decades, with advancements in preventive strategies, control of transplant-related complications, and preparative regimens. A risk class-based transplantation approach results in disease-free survival probabilities of 90%, 84%, and 78% for class 1, 2, and 3 thalassemia patients, respectively. Because of disease advancement, adult thalassemia patients have a higher risk for transplant-related toxicity and a 65% cure rate. Patients without matched donors could benefit from haploidentical mother-to-child transplantation. There is a high cure rate for children with SCA who receive HSCT following myeloablative conditioning protocols. Novel non-myeloablative transplantation protocols could make HSCT available to adult SCA patients who were previously excluded from allogeneic stem cell transplantation.
MeSH term(s)	Adult ; Anemia, Sickle Cell/therapy ; Graft vs Host Disease/etiology ; Hematopoietic Stem Cell Transplantation/methods ; Humans ; Living Donors ; Myeloablative Agonists/therapeutic use ; Thalassemia/therapy
Chemical Substances	Myeloablative Agonists
Language	English
Publishing date	2012-04-09
Publishing country	United States
Document type	Journal Article ; Review
ISSN	2157-1422
ISSN (online)	2157-1422
DOI	10.1101/cshperspect.a011825
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Article ; Online: Allogeneic hematopoietic stem cell transplantation from sibling and unrelated donors in pediatric patients with sickle cell disease-A single center experience.

Kogel, Friederike / Hakimeh, Dani / Sodani, Pietro / Lang, Peter / Kühl, Jörn-Sven / Hundsdoerfer, Patrick / Künkele, Annette / Eggert, Angelika / Oevermann, Lena / Schulte, Johannes H

Pediatric transplantation

2020 Volume 25, Issue 2, Page(s) e13892

Abstract: HSCT is curative in SCD. Patients with HLA-identical sibling donor have an excellent outcome ranging from 90%-100% overall and event-free survival. However, due to the lack of matched sibling donors this option is out of reach for 70% of patients with ... ...

Abstract	HSCT is curative in SCD. Patients with HLA-identical sibling donor have an excellent outcome ranging from 90%-100% overall and event-free survival. However, due to the lack of matched sibling donors this option is out of reach for 70% of patients with SCD. The pool of potential donors needs to be extended. Transplantations from HLA-matched unrelated donors were reported to be less successful with shorter event-free survival and higher incidences of complications including graft-vs-host disease, especially in patients with advanced stage SCD. Here we report transplantation outcomes for 25 children with SCD transplanted using HLA-matched grafts from related or unrelated donors. Overall survival was 100% with no severe (grade III-IV) graft-vs-host disease and a 12% rejection rate. Mixed donor chimerisms only occurred in transplantations from siblings, while transplantations from unrelated donors resulted in either complete donor chimerism or rejection. Despite the small patient number, overall and disease-free survival for unrelated donor transplantations is excellent in this cohort. The advanced disease state, higher alloreactive effect and stronger immunosuppression in unrelated donor transplantations raises patient risk, for which possible solutions could be found in optimization of transplant preparation, graft manipulation or haploidentical transplantation using T cell receptor α/β-depleted grafts.
MeSH term(s)	Adolescent ; Anemia, Sickle Cell/blood ; Anemia, Sickle Cell/immunology ; Anemia, Sickle Cell/mortality ; Anemia, Sickle Cell/therapy ; Biomarkers/blood ; Child ; Child, Preschool ; Chimerism ; Disease-Free Survival ; Female ; Follow-Up Studies ; Graft Rejection/diagnosis ; Graft Rejection/epidemiology ; Graft Rejection/immunology ; Graft Rejection/prevention & control ; Graft vs Host Disease/diagnosis ; Graft vs Host Disease/epidemiology ; Graft vs Host Disease/immunology ; Graft vs Host Disease/prevention & control ; HLA Antigens/immunology ; Hematopoietic Stem Cell Transplantation/methods ; Histocompatibility Testing ; Humans ; Infant ; Isoantibodies/blood ; Isoantibodies/immunology ; Male ; Retrospective Studies ; Siblings ; Transplantation, Homologous/methods ; Treatment Outcome ; Unrelated Donors ; Young Adult
Chemical Substances	Biomarkers ; HLA Antigens ; Isoantibodies
Language	English
Publishing date	2020-10-24
Publishing country	Denmark
Document type	Journal Article
ZDB-ID	1390284-2
ISSN	1399-3046 ; 1397-3142
ISSN (online)	1399-3046
ISSN	1397-3142
DOI	10.1111/petr.13892
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Zs.A 4947: Show issues

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Article: Successful recovery from COVID-19 pneumonia after receiving baricitinib, tocilizumab, and remdesivir. A case report: Review of treatments and clinical role of computed tomography analysis.

Sodani, Pietro / Mucci, Luciano / Girolimetti, Rita / Tedesco, Silvia / Monaco, Francesca / Campanozzi, Daniele / Brunori, Marino / Maltoni, Stefania / Bedetta, Samuele / Di Carlo, Anna M / Candoli, Piero / Mancini, Mauro / Rebonato, Alberto / D'Adamo, Francesca / Capalbo, Maria / Frausini, Gabriele

Respiratory medicine case reports

2020 Volume 31, Page(s) 101115

Abstract: The novel coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has caused a pandemic, threatening global public health. In the current paper, we describe our successful treatment of one COVID-19 ... ...

Abstract	The novel coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has caused a pandemic, threatening global public health. In the current paper, we describe our successful treatment of one COVID-19 pneumonia patient case with high mortality risk factors. Our experience underlines the importance of the use of a multidisciplinary therapeutic approach in order to achieve a favorable clinical outcome. Further, enhancing the capability of the COVID-19 diagnosis with the use of the chest imaging modalities is discussed.
Keywords	covid19
Language	English
Publishing date	2020-06-27
Publishing country	England
Document type	Case Reports
ZDB-ID	2666110-X
ISSN	2213-0071
ISSN	2213-0071
DOI	10.1016/j.rmcr.2020.101115
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Article ; Online: Progress in hematopoietic stem cell transplantation as allogeneic cellular gene therapy in thalassemia.

Isgrò, Antonella / Gaziev, Javid / Sodani, Pietro / Lucarelli, Guido

Annals of the New York Academy of Sciences

2010 Volume 1202, Page(s) 149–154

Abstract: Allogeneic hemopoietic stem cell transplantation (HSCT) represents one of the best cures for thalassemia. Currently, HSCT for thalassemia consists of allogeneic stem cell gene therapy and still awaits autologous genetically modified stem cell ... ...

Abstract	Allogeneic hemopoietic stem cell transplantation (HSCT) represents one of the best cures for thalassemia. Currently, HSCT for thalassemia consists of allogeneic stem cell gene therapy and still awaits autologous genetically modified stem cell transplantation. HSCT for thalassemia has substantially improved over the last two decades, due in large part to improvements in preventive strategies, the effective control of transplant-related complications, and the development of new preparative regimens. A risk classes-based approach to transplantation in thalassemia has led to disease-free survival probability of 87, 85, and 80% in classes 1, 2, and 3 patients, respectively. Adult thalassemia patients, who are higher risk patients for transplant-related toxicity due to an advanced phase of the disease, have a cure rate of 65% with current treatment protocol. Patients who do not have matched family or unrelated donors could benefit from haploidentical mother-to-child transplantation. Overall, the results of this type of transplantation appear encouraging.
MeSH term(s)	Adult ; Bone Marrow Transplantation ; Child ; Disease-Free Survival ; Genetic Therapy ; HLA Antigens ; Hematopoietic Stem Cell Transplantation ; Humans ; Thalassemia/physiopathology ; Thalassemia/therapy ; Tissue Donors ; Transplantation Conditioning ; Transplantation, Homologous
Chemical Substances	HLA Antigens
Language	English
Publishing date	2010-08
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	211003-9
ISSN	1749-6632 ; 0077-8923
ISSN (online)	1749-6632
ISSN	0077-8923
DOI	10.1111/j.1749-6632.2010.05543.x
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Article ; Online: Haploidentical HSCT for hemoglobinopathies: improved outcomes with TCRαβ

Gaziev, Javid / Isgrò, Antonella / Sodani, Pietro / Paciaroni, Katia / De Angelis, Gioia / Marziali, Marco / Ribersani, Michela / Alfieri, Cecilia / Lanti, Alessandro / Galluccio, Tiziana / Adorno, Gaspare / Andreani, Marco

Blood advances

2018 Volume 2, Issue 3, Page(s) 263–270

Abstract: We examined outcomes of haploidentical hematopoietic cell transplantation (haplo-HCT) using T-cell receptor ... ...

Abstract	We examined outcomes of haploidentical hematopoietic cell transplantation (haplo-HCT) using T-cell receptor αβ
MeSH term(s)	Adolescent ; Antigens, CD19 ; Antigens, CD34 ; Child ; Child, Preschool ; Graft Rejection ; Graft Survival/immunology ; Graft vs Host Disease/etiology ; Hematopoietic Stem Cell Transplantation/adverse effects ; Hematopoietic Stem Cell Transplantation/methods ; Hemoglobinopathies/complications ; Hemoglobinopathies/mortality ; Hemoglobinopathies/therapy ; Humans ; Lymphocyte Depletion/methods ; Male ; Receptors, Antigen, T-Cell, alpha-beta ; Retrospective Studies ; Survival Analysis ; Transplantation, Haploidentical/adverse effects ; Transplantation, Haploidentical/methods ; Treatment Outcome
Chemical Substances	Antigens, CD19 ; Antigens, CD34 ; Receptors, Antigen, T-Cell, alpha-beta
Language	English
Publishing date	2018-02-05
Publishing country	United States
Document type	Journal Article
ZDB-ID	2876449-3
ISSN	2473-9537 ; 2473-9529
ISSN (online)	2473-9537
ISSN	2473-9529
DOI	10.1182/bloodadvances.2017012005
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Article: Impact of Donor-Specific anti-HLA Antibodies and Donor KIR Characteristics in Haploidentical HSCT for Beta-Thalassemia.

Andreani, Marco / Testi, Manuela / Sodani, Pietro / Troiano, Maria / Di Luzio, Andrea / Testa, Giuseppe / Falco, Michela / Poggi, Elvira / Gaziev, Javid / Piazza, Antonina

Mediterranean journal of hematology and infectious diseases

2017 Volume 9, Issue 1, Page(s) e2017020

Language	English
Publishing date	2017-03-01
Publishing country	Italy
Document type	Journal Article
ZDB-ID	2674750-9
ISSN	2035-3006
ISSN	2035-3006
DOI	10.4084/MJHID.2017.020
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Article ; Online: Successful recovery from COVID-19 pneumonia after receiving baricitinib, tocilizumab, and remdesivir. A case report

Pietro Sodani / Luciano Mucci / Rita Girolimetti / Silvia Tedesco / Francesca Monaco / Daniele Campanozzi / Marino Brunori / Stefania Maltoni / Samuele Bedetta / Anna M. Di Carlo / Piero Candoli / Mauro Mancini / Alberto Rebonato / Francesca D'Adamo / Maria Capalbo / Gabriele Frausini

Respiratory Medicine Case Reports, Vol 31, Iss , Pp 101115- (2020)

Review of treatments and clinical role of computed tomography analysis

2020

Abstract	The novel coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has caused a pandemic, threatening global public health. In the current paper, we describe our successful treatment of one COVID-19 pneumonia patient case with high mortality risk factors. Our experience underlines the importance of the use of a multidisciplinary therapeutic approach in order to achieve a favorable clinical outcome. Further, enhancing the capability of the COVID-19 diagnosis with the use of the chest imaging modalities is discussed.
Keywords	COVID-19 ; SARS-CoV-2 ; Coronavirus ; Viral pneumonia ; Diseases of the respiratory system ; RC705-779 ; covid19
Language	English
Publishing date	2020-01-01T00:00:00Z
Publisher	Elsevier
Document type	Article ; Online
Database	BASE - Bielefeld Academic Search Engine (life sciences selection)

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