Research

10 pages, 1904 KiB

Open AccessArticle

Busulfan/Cyclophosphamide Compared with Melphalan as a Conditioning Regimen for Autologous Transplantation of Multiple Myeloma: A Long-Term Assessment

by Shiyuan Zhou, Yingying Zhai, Lingzhi Yan, Xiaolan Shi, Jingjing Shang, Depei Wu, Chengcheng Fu and Song Jin

J. Clin. Med. 2023, 12(19), 6239; https://doi.org/10.3390/jcm12196239 - 27 Sep 2023

Viewed by 967

Abstract

Background: Melphalan was poorly available in mainland China. The aim of this study is to explore the dose-adjusted busulfan/cyclophosphamide (BU/CY) as an alternative regimen in auto stem cell transplantation (ASCT) for multiple myeloma (MM). Methods: A total of 105 newly diagnosed MM patients [...] Read more.

Background: Melphalan was poorly available in mainland China. The aim of this study is to explore the dose-adjusted busulfan/cyclophosphamide (BU/CY) as an alternative regimen in auto stem cell transplantation (ASCT) for multiple myeloma (MM). Methods: A total of 105 newly diagnosed MM patients undergoing ASCT during May 2012 and August 2017 were retrospectively analyzed. The BU/CY regimen was applied to 64 patients. Busulfan (9.6 mg/kg or 8.0 mg/kg in total) and cyclophosphamide (3.6 g/m² or 3.0 g/m² in total) were administered according to the creatinine clearance rate (CCR). A high-dose melphalan (HDMEL) regimen (200 mg/m²) was given to the other 41 patients. Results: At a median follow-up of 65 (1~119) months, estimated overall survival (OS) and progression-free survival (PFS) at 104 months in the BU/CY and HDMEL groups were 35.6% vs. 20.5% (p = 0.263) and 20.2% vs. 2.4% (p = 0.035), respectively. The median overall survival (OS) and PFS of the HDMEL and BU/CY groups were 55 vs. 70.5 months and 26 vs. 46.5 months, respectively. In multivariate analysis, the BU/CY regimen was found to be the only protective factor for PFS. No lethal toxicity was found in the BU/CY group, and treatment-related mortality (TRM) in 100 days was similar to the HDMEL group. Conclusions: MM patients may also benefit from the dose-adjusted BU/CY regimen. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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12 pages, 1428 KiB

Open AccessArticle

Handheld Ultrasound or Conventional Ultrasound Devices in Patients Undergoing HCT: A Validation Study

by Andrea Duminuco, Alessandra Cupri, Rosario Massimino, Salvatore Leotta, Giulio Antonio Milone, Bruno Garibaldi, Giulia Giuffrida, Orazio Garretto and Giuseppe Milone

J. Clin. Med. 2023, 12(2), 520; https://doi.org/10.3390/jcm12020520 - 08 Jan 2023

Cited by 2 | Viewed by 1647

Abstract

Abdominal ultrasound exams play a major role in the diagnosis of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD). The development of portable hand-held ultrasound devices (HHUS) has been shown to facilitate the diagnosis of many diseases, but little data on the value of HHUS in [...] Read more.

Abdominal ultrasound exams play a major role in the diagnosis of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD). The development of portable hand-held ultrasound devices (HHUS) has been shown to facilitate the diagnosis of many diseases, but little data on the value of HHUS in the diagnosis of SOS/VOD are available. We performed a study aimed at validating portable ultrasound (US) devices in the setting of hematopoietic stem cell transplant (HCT). Sixteen evaluable patients undergoing allogeneic HCT were studied using conventional US and HHUS during the first 3 weeks after transplant. The results obtained demonstrate that there is a close correlation between conventional and handheld ultrasound examination in the measurement of the right hepatic lobe (r = 0.912, p < 0.0001), the left hepatic lobe (r = 0.843, p < 0.0001), the portal vein (PV) (r = 0.724, p < 0.0001), and the spleen (r = 0.983, p < 0.0001) based on Pearson’s correlation. The same data, analyzed through Lin’s concordance correlation coefficient, evidenced a substantial level of agreement in the comparison of the spleen and right hepatic lobe, while a lower grade of agreement in the measurement of the portal vein and left hepatic lobe. Moreover, there was good agreement between results obtained by the two types of ultrasound devices in assessing ascites (p < 0.0001), gallbladder thickening (p < 0.0001), and the direction of PV flow (p < 0.0001). HHUS device allows the study of HokUs-10 parameters with an excellent agreement with conventional US, and may contribute to SOS/VOD diagnosis. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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10 pages, 810 KiB

Open AccessArticle

Impact of Cryopreservation of Peripheral Blood Stem Cells (PBSC) in Transplantation from Matched Unrelated Donor (MUD)

by Gabriele Facchin, Chiara Savignano, Marta Lisa Battista, Miriam Isola, Maria De Martino, Giuseppe Petruzzellis, Chiara Rosignoli, Umberto Pizzano, Michela Cerno, Giulia De Cecco, Antonella Bertone, Giovanni Barillari, Renato Fanin and Francesca Patriarca

J. Clin. Med. 2022, 11(14), 4114; https://doi.org/10.3390/jcm11144114 - 15 Jul 2022

Cited by 6 | Viewed by 1700

Abstract

Background: Cryopreservation of PBSC for allogenic hematopoietic stem cell transplantation (allo-HSCT) was implemented due to the current Coronavirus 2019 pandemic. The impact of match unrelated donor (MUD) graft freezing on the outcome of allo-HSCT in terms of hematological recovery, graft versus host [...] Read more.

Background: Cryopreservation of PBSC for allogenic hematopoietic stem cell transplantation (allo-HSCT) was implemented due to the current Coronavirus 2019 pandemic. The impact of match unrelated donor (MUD) graft freezing on the outcome of allo-HSCT in terms of hematological recovery, graft versus host disease (GVHD), and survival are still controversial. Methods: In this study, we compared graft composition, clinical characteristics, and outcome of 31 allo-HSCT from MUD cryopreserved PBSC (Cryo Group) with 23 matched-pair allo-HSCT from fresh MUD PBSC (Fresh Group) performed in our center between January 2020 and July 2021. Results: No significant differences were recognized in clinical characteristics of patients, donors, and transplants between the Cryo and Fresh groups except for a better prognostic comorbidity index (HCT-CI) of the Cryo group. In the Cryo Group, the median time from apheresis to cryopreservation was 46.0 h (range 23.8–53.5), while the median time from cells collection and reinfusion was 13.9 days (range 5.8–28.1). In the Fresh Group, median time from apheresis to reinfusion was 35.6 h (range 21.4–51.2). The number of viable (7-AAD negative) CD34+ cells per kg patient infused was significantly lower in the Cryo Group (5.2 ± 1.9 × 10⁶/kg vs. 7.0 ± 1.3 × 10⁶/kg; p < 0.001). Indeed, there was a 36% (11–70) median loss of viable CD34+/kg cells after freezing. All patients engrafted: median time to neutrophil engraftment (>0.5 × 10⁹/L) was 13.5 days (range 12–15) for Cryo Group and 14 days (range 13–16) days for Fresh Group (p = 0.522), while the median time to platelet engraftment (>20 × 10⁹/L) was, respectively, 14 (range 12–18) and 15 (range 12–17) days (p = 0.904). The incidence of grade ≥ 2 acute GVHD was similar in the two groups (56.5% Cryo Group vs. 60.0% Fresh Group; p = 0.832) and no differences in terms of OS (p = 0.090), PFS (p = 0.200) and TRM (p = 0.970) were observed between the Cryo and Fresh groups. Conclusions: In our series, no differences between the Cryo and Fresh groups were found in engraftment, grade ≥ 2 acute GVHD incidence, OS, PFS, and TRM despite a lower CD34+ infused dose in the Cryo Group. Frozen PBSCs could be considered a safe option also for allo-HSCT from MUD but a higher amount of PBSC should be collected to warrant an adequate viable CD34+ post-thawing. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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10 pages, 266 KiB

Open AccessArticle

BeEAM High-Dose Chemotherapy with Polatuzumab (Pola-BeEAM) before ASCT in Patients with DLBCL—A Pilot Study

by Tanja Stoffel, Ulrike Bacher, Yara Banz, Michael Daskalakis, Urban Novak and Thomas Pabst

J. Clin. Med. 2022, 11(13), 3748; https://doi.org/10.3390/jcm11133748 - 28 Jun 2022

Cited by 1 | Viewed by 1754

Abstract

(1) Introduction: BEAM is a high-dose chemotherapy (HDCT) frequently administered before autologous stem cell transplantation (ASCT) in diffuse large B-cell lymphoma (DLBCL). Bendamustine replacing BCNU (BeEAM) is similarly effective at lower toxicities. However, relapse remains the major cause of death in DLBCL. (2) [...] Read more.

(1) Introduction: BEAM is a high-dose chemotherapy (HDCT) frequently administered before autologous stem cell transplantation (ASCT) in diffuse large B-cell lymphoma (DLBCL). Bendamustine replacing BCNU (BeEAM) is similarly effective at lower toxicities. However, relapse remains the major cause of death in DLBCL. (2) Methods: This is a 12-patient pilot study of the BeEAM preparative regimen with additional polatuzumab vedotin (PV, targeting CD79b) aiming to establish feasibility and to reduce toxicity without increasing the early progression rate. PV was given once at the standard dose of 1.8 mg/kg at day −6 together with BeEAM-HDCT (days −7 to −1) before ASCT. (3) Results: 8/12 patients (67%) received PV with BeEAM as a consolidation of first-line treatment, and 4/12 patients (33%) received PV with BeEAM after relapse treatment. All patients experienced complete engraftment (neutrophils: median 11 days; platelets: 13 days). Gastrointestinal toxicities occurred in 7/12 patients (58%, grade 3). All patients developed neutropenic infections with at least one identified pathogen (bacterial: 10/12 patients; viral: 2/12; and fungal: 1/12). The complete remission rate by PET-CT 100 days post-ASCT was 92%, with one mortality due to early progression. Eleven out of twelve patients (92%) were alive without progression after a median follow-up of 15 months. (4) Conclusions: Our study with 12 patients suggests that combining PV with BeEAM HDCT is feasible and safe, but the limited cohort prevents definite conclusions regarding efficacy. Larger cohorts must be evaluated. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

9 pages, 439 KiB

Open AccessArticle

Feasibility of Outpatient Stem Cell Transplantation in Multiple Myeloma and Risk Factors Predictive of Hospital Admission

by Kristin Larsen, Horace Spencer, Meera Mohan, Clyde Bailey, Kerri Hill, Mathew Kottarathara, Richa Parikh, Shadiqul Hoque, Amani Erra, Angel A. Mitma, Pankaj Mathur, Lakshmi Yarlagadda, Sravani Gundarlapalli, Yetunde Ogunsesan, Munawwar Hussain, Nishanth Thalambedu, Jaskirat Sethi, Samer Al Hadidi, Sharmilan Thanendrarajan, Monica Graziutti, Maurizio Zangari, Bart Barlogie, Frits van Rhee, Guido Tricot and Carolina Schinke Show full author list Hide full author list

J. Clin. Med. 2022, 11(6), 1640; https://doi.org/10.3390/jcm11061640 - 16 Mar 2022

Cited by 4 | Viewed by 2167

Abstract

High-dose chemotherapy followed by autologous stem cell transplantation (ASCT) remains the standard of care for multiple myeloma (MM) patients. Although outpatient ASCT has been shown to be safe and feasible, the procedure is overall rare with most patients in the US undergoing inpatient [...] Read more.

High-dose chemotherapy followed by autologous stem cell transplantation (ASCT) remains the standard of care for multiple myeloma (MM) patients. Although outpatient ASCT has been shown to be safe and feasible, the procedure is overall rare with most patients in the US undergoing inpatient ASCT. Furthermore, hospitalization rates for patients that undergo outpatient ASCT remain high. Adequate markers that predict hospitalization during outpatient ASCT are lacking, yet would be of great clinical value to select patients that are suited to outpatient ASCT. In this study we aimed to elucidate differences between planned outpatient and inpatient ASCT and further evaluated clinical characteristics that are significantly associated with hospitalization during planned outpatient hospitalization. Factors that were significantly associated with a planned inpatient ASCT included an advanced MM disease stage, worse performance status as well as non-Caucasian race, while low albumin levels and female gender were significantly associated with hospitalization during outpatient ASCT. The results of this analysis provide crucial knowledge of factors that are associated with planned inpatient ASCT and hospitalization during outpatient ASCT and could guide the treating physician in decision-making and further facilitate outpatient transplantation. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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11 pages, 432 KiB

Open AccessArticle

Addition of a Single Low Dose of Anti T-Lymphocyte Globulin to Post-Transplant Cyclophosphamide after Allogeneic Hematopoietic Stem Cell Transplant: A Pilot Study

by Elisabetta Xue, Francesca Lorentino, Maria Teresa Lupo Stanghellini, Fabio Giglio, Simona Piemontese, Daniela Teresa Clerici, Francesca Farina, Sara Mastaglio, Alessandro Bruno, Edoardo Campodonico, Rosamaria Nitti, Magda Marcatti, Andrea Assanelli, Consuelo Corti, Fabio Ciceri, Jacopo Peccatori and Raffaella Greco

J. Clin. Med. 2022, 11(4), 1106; https://doi.org/10.3390/jcm11041106 - 19 Feb 2022

Cited by 8 | Viewed by 2059

Abstract

Correlation between risk of graft-versus-host disease (GvHD) and CD3⁺ counts within the peripheral blood stem cell graft has recently been reported in the setting of post-transplant cyclophosphamide (PT-Cy). We aimed to investigate the benefit of the addition of a single dose of [...] Read more.

Correlation between risk of graft-versus-host disease (GvHD) and CD3⁺ counts within the peripheral blood stem cell graft has recently been reported in the setting of post-transplant cyclophosphamide (PT-Cy). We aimed to investigate the benefit of the addition of a single dose of anti-T lymphocyte globulin (ATLG 5 mg/kg) to PT-Cy in this setting. Starting in 2019, all patients receiving PBSC transplant containing CD3⁺ counts above 300 × 10⁶/kg (study group) received a post-transplant dose of ATLG in addition to standard PT-Cy. The study was designed as a real-life analysis and included all consecutive Hematopoietic Stem Cell Transplantation (HSCT) recipients according to the above-mentioned inclusion criterion (n = 21), excluding cord blood and bone marrow donors. Using a 1:2 matched-pair analysis, we compared the outcomes with a historical population who received PT-Cy only (control group). We found a delayed platelet engraftment (29% vs. 45% at 30 days, p = 0.03) and a non-significant trend toward higher risk of poor graft function (29% vs. 19%, p = 0.52). The addition of ATLG impacted long-term immune reconstitution on the CD4⁺ subsets, but this did not translate into higher rate of relapse or viral infection. Acute GvHD was not significantly impacted, but 1-year cumulative incidence of chronic GvHD was significantly lower in the study group (15% vs. 41%, p = 0.04). Survival outcomes were comparable. In conclusion PT-Cy and ATLG was overall safe and translated into a low rate of chronic GvHD incidence. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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12 pages, 272 KiB

Open AccessArticle

C-Reactive Protein Monitoring and Clinical Presentation of Fever as Predictive Factors of Prolonged Febrile Neutropenia and Blood Culture Positivity after Autologous Hematopoietic Stem Cell Transplantation—Single-Center Real-Life Experience

by Daniela Carcò, Uros Markovic, Paolo Castorina, Valeria Iachelli, Tecla Pace, Paola Guardo, Gabriella Amato, Federica Galbo, Paola Scirè and Gaetano Moschetti

J. Clin. Med. 2022, 11(2), 312; https://doi.org/10.3390/jcm11020312 - 09 Jan 2022

Viewed by 1422

Abstract

Background: Febrile neutropenia (FN) is a medical emergency that requires urgent evaluation, timely administration of empiric broad-spectrum antibiotics and careful monitoring in order to optimize the patient’s outcome, especially in the setting of both allogeneic and autologous hematopoietic stem cell transplant (ASCT). Methods: [...] Read more.

Background: Febrile neutropenia (FN) is a medical emergency that requires urgent evaluation, timely administration of empiric broad-spectrum antibiotics and careful monitoring in order to optimize the patient’s outcome, especially in the setting of both allogeneic and autologous hematopoietic stem cell transplant (ASCT). Methods: In this real-life retrospective study, a total of 49 consecutive episodes of FN were evaluated in 40 adult patients affected by either multiple myeloma (thirty-eight) or lymphoma (eleven), following ASCT, with nine patients having fever in both of the tandem transplantations. Results: Febrile neutropenia occurred a median of 7 days from ASCT. Median duration of FN was 2 days, with 25% of population that had fever for at least four days. Ten patients had at least one fever spike superior to 39 °C, while the median number of daily fever spikes was two. Twenty patients had positive blood cultures with XDR germs, namely Pseudomonas aeruginosa and Klebsiella pneumoniae, present in seven cases. ROC analysis of peak C-reactive protein (CRP) values was conducted based on blood culture positivity and a value of 12 mg/dL resulted significant. Onset of prolonged fever with a duration greater than 3 days was associated with the presence of both a peak number of three or more daily fever spikes (p = 0.02) and a body temperature greater than 39 °C (p = 0.04) based on odds ratio (OR). Blood culture positivity and peak CRP values greater than 12 mg/dL were also associated with prolonged fever duration, p = 0.04, and p = 0.03, respectively. The probability of blood culture positivity was also greater in association with fever greater than 39 °C (p = 0.04). Furthermore, peak CRP values below the cut-off showed less probability of positive blood culture (p = 0.02). Conclusions: In our study, clinical characteristics of fever along with peak CRP levels were associated with a higher probability of both prolonged fever duration and positive blood culture, needing extended antibiotic therapy. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

Review

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12 pages, 308 KiB

Open AccessReview

Human Leucocyte Antigen System and Selection of Unrelated Hematopoietic Stem Cell Donors: Impact of Patient–Donor (Mis)matching and New Challenges with the Current Technologies

by Roberto Crocchiolo and Gianni Rombolà

J. Clin. Med. 2023, 12(2), 646; https://doi.org/10.3390/jcm12020646 - 13 Jan 2023

Cited by 2 | Viewed by 1414

Abstract

The selection of hematopoietic stem cell donors for allogeneic transplantation (allo-HSCT) is mainly driven by human leucocyte antigen (HLA) matching between patient and donor, with HLA-identical matched siblings being the preferred choice in most situations. Although other clinical and demographical variables matter, especially, [...] Read more.

The selection of hematopoietic stem cell donors for allogeneic transplantation (allo-HSCT) is mainly driven by human leucocyte antigen (HLA) matching between patient and donor, with HLA-identical matched siblings being the preferred choice in most situations. Although other clinical and demographical variables matter, especially, donor age, which is unequivocally associated with better transplant outcomes, the histocompatibility criteria have a central role in the search for the best donor, particularly in the setting of unrelated allo-HSCT where HLA disparities between patient and donor are frequent. The present review is focused on the role of HLA incompatibilities on patient outcome according to the most recent literature, in an attempt to guide transplant physicians and search coordinators during the process of adult unrelated-donor selection. The technological progresses in HLA typing, i.e., with next-generation sequencing (NGS), now allow disclosing a growing number of HLA incompatibilities associated with a heterogeneous and sometimes unknown spectrum of clinical severity. Their immunogenic characteristics, i.e., their position inside or outside the antigen recognition domain (ARD), their permissiveness, their intronic or exonic nature and even the expected expression of the HLA loci where those mismatches occur, will be presented and discussed here, integrating the advances in the immunobiology of transplantation with survival and toxicity outcomes reported in the most relevant studies, within the perspective of improving donor selection in the current practice. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

12 pages, 1956 KiB

Open AccessReview

Post-Transplant Lymphoproliferative Disease (PTLD) after Allogeneic Hematopoietic Stem Cell Transplantation: Biology and Treatment Options

by Michele Clerico, Irene Dogliotti, Andrea Aroldi, Chiara Consoli, Luisa Giaccone, Benedetto Bruno and Federica Cavallo

J. Clin. Med. 2022, 11(24), 7542; https://doi.org/10.3390/jcm11247542 - 19 Dec 2022

Cited by 8 | Viewed by 2263

Abstract

Post-transplant lymphoproliferative disease (PTLD) is a serious complication occurring as a consequence of immunosuppression in the setting of allogeneic hematopoietic stem cell transplantation (alloHSCT) or solid organ transplantation (SOT). The majority of PTLD arises from B-cells, and Epstein–Barr virus (EBV) infection is present [...] Read more.

Post-transplant lymphoproliferative disease (PTLD) is a serious complication occurring as a consequence of immunosuppression in the setting of allogeneic hematopoietic stem cell transplantation (alloHSCT) or solid organ transplantation (SOT). The majority of PTLD arises from B-cells, and Epstein–Barr virus (EBV) infection is present in 60–80% of the cases, revealing the central role played by the latent infection in the pathogenesis of the disease. Therefore, EBV serological status is considered the most important risk factor associated with PTLDs, together with the depth of T-cell immunosuppression pre- and post-transplant. However, despite the advances in pathogenesis understanding and the introduction of novel treatment options, PTLD arising after alloHSCT remains a particularly challenging disease, and there is a need for consensus on how to treat rituximab-refractory cases. This review aims to explore the pathogenesis, risk factors, and treatment options of PTLD in the alloHSCT setting, finally focusing on adoptive immunotherapy options, namely EBV-specific cytotoxic T-lymphocytes (EBV-CTL) and chimeric antigen receptor T-cells (CAR T). Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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9 pages, 268 KiB

Open AccessReview

Thrombocytopenia and Therapeutic Strategies after Allogeneic Hematopoietic Stem Cell Transplantation

by Leyre Bento, Mariana Canaro, José María Bastida and Antonia Sampol

J. Clin. Med. 2022, 11(5), 1364; https://doi.org/10.3390/jcm11051364 - 02 Mar 2022

Cited by 6 | Viewed by 2324

Abstract

Thrombocytopenia following allogeneic hematopoietic stem cell transplantation is a usual complication and can lead to high morbidity and mortality. New strategies, such as the use of another graft versus host-disease prophylaxis, alternative donors, and management of infections, have improved the survival of these [...] Read more.

Thrombocytopenia following allogeneic hematopoietic stem cell transplantation is a usual complication and can lead to high morbidity and mortality. New strategies, such as the use of another graft versus host-disease prophylaxis, alternative donors, and management of infections, have improved the survival of these patients. The mechanisms are unknown; therefore, the identification of new strategies to manage this potentially serious problem is needed. Thrombopoietin receptor agonists are currently available to stimulate platelet production. Some small retrospective studies have reported their potential efficacy in an allogeneic stem cell transplant setting, confirming good tolerability. Recent studies with higher numbers of patients also support their safety and efficacy in this setting, hence establishing the use of these drugs as a promising strategy for this post-transplant complication. However, prospective trials are needed to confirm these results. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

26 pages, 5316 KiB

Open AccessReview

Autologous Hematopoietic Stem Cell Transplantation in Multiple Sclerosis Patients: Monocentric Case Series and Systematic Review of the Literature

by Francesco Patti, Clara Grazia Chisari, Simona Toscano, Sebastiano Arena, Chiara Finocchiaro, Vincenzo Cimino and Giuseppe Milone

J. Clin. Med. 2022, 11(4), 942; https://doi.org/10.3390/jcm11040942 - 11 Feb 2022

Cited by 5 | Viewed by 3213

Abstract

Multiple sclerosis (MS) is a chronic, inflammatory and immune-mediated disease of the central nervous system (CNS), commonly affecting young adults and potentially associated with life-long disability. About 14 disease-modifying treatments (DMTs) are currently approved for the treatment of MS. However, despite the use [...] Read more.

Multiple sclerosis (MS) is a chronic, inflammatory and immune-mediated disease of the central nervous system (CNS), commonly affecting young adults and potentially associated with life-long disability. About 14 disease-modifying treatments (DMTs) are currently approved for the treatment of MS. However, despite the use of highly effective therapies, some patients exhibit a highly active disease with an aggressive course from onset and a higher risk of long-term disability accrual. In the last few years, several retrospective studies, clinical trials, meta-analyses and systematic reviews have investigated autologous hematopoietic stem cell transplantation (AHSCT) as a possible therapeutic option in order to address this unmet clinical need. These studies demonstrated that AHSCT is a highly efficacious and relatively safe therapeutic option for the treatment of highly active MS. Particularly, over recent years, the amount of evidence has grown, with significant improvements in the development of patient selection criteria, choice of the most suitable transplant technique and clinical experience. In this paper, we present six patients who received AHSCT in our MS center and we systematically reviewed recent evidence about the long-term efficacy and safety of AHSCT and the placement of AHSCT in the rapidly evolving therapeutic armamentarium for MS. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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16 pages, 1119 KiB

Open AccessReview

Conditioning Regimens in Patients with β-Thalassemia Who Underwent Hematopoietic Stem Cell Transplantation: A Scoping Review

by Olga Mulas, Brunella Mola, Giovanni Caocci and Giorgio La Nasa

J. Clin. Med. 2022, 11(4), 907; https://doi.org/10.3390/jcm11040907 - 09 Feb 2022

Cited by 10 | Viewed by 3796

Abstract

The success of transplant procedures in patients with beta-thalassemia major (β-thalassemia) goes hand-in-hand with improvements in disease knowledge, better supportive care, discoveries in immunogenetics, increase in stem cell sources, and enhancement of conditioning regimens. The aim of this scoping review was to report [...] Read more.

The success of transplant procedures in patients with beta-thalassemia major (β-thalassemia) goes hand-in-hand with improvements in disease knowledge, better supportive care, discoveries in immunogenetics, increase in stem cell sources, and enhancement of conditioning regimens. The aim of this scoping review was to report the evolution of conditioning regimes for β-thalassemia hematopoietic stem cell transplantation. We performed a systematic search for all relevant articles published before July 2021, using the following Medical Subject Headings: “bone marrow transplantation”, “stem cell transplantation”, “allogeneic”, “thalassemia”, “β-thalassemia”, and “thalassemia major”. The final analysis included 52 studies, published between 1988 and 2021, out of 3877 records. The most common conditioning regimen was a combination of busulfan and cyclophosphamide, with successive dose adjustments or remodulation based on patient characteristics. Pre-transplant treatments, reductions in cyclophosphamide dosage, or the adoption of novel agents such as treosulphan all improved overall survival and thalassemia-free survival in transplant-related mortality high-risk patients. Conditioning regimes were modulated for those without a suitable fully matched sibling or unrelated donor, with encouraging results. Hematopoietic stem cell transplantation with haploidentical donors is currently available to virtually all patients with β-thalassemia. However, disparities in outcome are still present around the world. In developing and limited-resource countries, where most diagnoses are focused, transplants are not always available. Therefore, more efforts are needed to close this treatment gap. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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29 pages, 5032 KiB

Open AccessReview

Endothelial Dysfunction after Hematopoietic Stem Cell Transplantation: A Review Based on Physiopathology

by Giuseppe Milone, Claudia Bellofiore, Salvatore Leotta, Giulio Antonio Milone, Alessandra Cupri, Andrea Duminuco, Bruno Garibaldi and Giuseppe Palumbo

J. Clin. Med. 2022, 11(3), 623; https://doi.org/10.3390/jcm11030623 - 26 Jan 2022

Cited by 18 | Viewed by 4786

Abstract

Endothelial dysfunction (ED) is frequently encountered in transplant medicine. ED is an argument of high complexity, and its understanding requires a wide spectrum of knowledge based on many fields of basic sciences such as molecular biology, immunology, and pathology. After hematopoietic stem cell [...] Read more.

Endothelial dysfunction (ED) is frequently encountered in transplant medicine. ED is an argument of high complexity, and its understanding requires a wide spectrum of knowledge based on many fields of basic sciences such as molecular biology, immunology, and pathology. After hematopoietic stem cell transplantation (HSCT), ED participates in the pathogenesis of various complications such as sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD), graft-versus-host disease (GVHD), transplant-associated thrombotic microangiopathy (TA-TMA), idiopathic pneumonia syndrome (IPS), capillary leak syndrome (CLS), and engraftment syndrome (ES). In the first part of the present manuscript, we briefly review some biological aspects of factors involved in ED: adhesion molecules, cytokines, Toll-like receptors, complement, angiopoietin-1, angiopoietin-2, thrombomodulin, high-mobility group B-1 protein, nitric oxide, glycocalyx, coagulation cascade. In the second part, we review the abnormalities of these factors found in the ED complications associated with HSCT. In the third part, a review of agents used in the treatment of ED after HSCT is presented. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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Other

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15 pages, 1109 KiB

Open AccessSystematic Review

Comparison of Different Rabbit Anti-Thymocyte Globulin Formulations in the Prophylaxis of Graft-Versus-Host Disease: A Systematic Review

by Jarosław Dybko, Ugo Giordano, Justyna Pilch, Jakub Mizera, Artur Borkowski and Monika Mordak-Domagała

J. Clin. Med. 2023, 12(17), 5449; https://doi.org/10.3390/jcm12175449 - 22 Aug 2023

Cited by 1 | Viewed by 1185

Abstract

Allogeneic hematopoietic stem cell transplantation (allo-HCT) is a potentially curative treatment modality, frequently used for patients suffering from haematological malignancies. In the last two decades, there have been multiple randomised controlled trials (RCTs), review articles, and meta-analyses addressing the efficacy of rabbit anti-thymocyte [...] Read more.

Allogeneic hematopoietic stem cell transplantation (allo-HCT) is a potentially curative treatment modality, frequently used for patients suffering from haematological malignancies. In the last two decades, there have been multiple randomised controlled trials (RCTs), review articles, and meta-analyses addressing the efficacy of rabbit anti-thymocyte globulin (r-ATG) as a graft-versus-host disease (GvHD) prophylaxis. Nevertheless, only a few aimed to compare the effectiveness of different r-ATG formulations. Since the last article we retrieved comparing different r-ATGs in GvHD prophylaxis dates back to 2017, we performed a systematic literature review of articles published since 2017 to this day, utilising PubMed, Scopus, Cochrane, and MEDLINE, with the main endpoints being prophylaxis of acute GvHD (aGvHD) and chronic GvHD (cGvHD). We subjected to scrutiny a total of five studies, of which four compared the differences between Thymoglobulin (ATG-T) and Grafalon (ATG-G), and one discussed the impact of ATG-T dose. Overall, cGvHD, aGvHD grades II–IV, TRM, OS, NRM, LFS, relapse, overall infections, and EBV reactivation do not seem to be affected by the type of utilised rATG. However, data on aGvHD grades III–IV, GRFS, moderate–severe cGvHD, and CMV reactivation is conflicting. Through our research, we sought to summarise the most recent findings concerning r-ATGs in allo-HCT, and provide insight into the differences between the targets and origin of various ATG formulations. Full article

(This article belongs to the Special Issue Hematopoietic Stem Cell Transplantation: Current Status and Future Research)

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