Publications by authors named "Damiano Rondelli"

The eighth workshop of the Worldwide Network for Blood and Marrow Transplantation (WBMT) was held in Islamabad, Pakistan, from September 22 to 23, 2022, aiming to foster hematopoietic stem cell transplant (HSCT) activity in the World Health Organization (WHO) Eastern Mediterranean Region (EMRO). Participating countries, including Pakistan, Oman, Iran, and Saudi Arabia, reported increased HSCT in the last few years, whereas others from the EMRO and beyond, including Qatar, United Arab Emirates, Nepal, and Bangladesh, started HSCT recently and have developed HSCT programs with excellent results. During educational sessions and open dialog, participating teams and international experts from the WBMT shared their experience and discussed minimum essential requirements for establishing and expanding HSCT in emerging countries, indications for HSCT training and dissemination of knowledge, stem cell donor selection and safety, quality assurance in transplant centers, and the value and importance of transplant outcome databases.

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  • Autologous peripheral blood stem cell (PBSC) transplantation is a common treatment for conditions like multiple myeloma and lymphomas, and it usually involves freezing the stem cells before transplant.* -
  • A recent systematic review of 19 transplant centers that performed non-cryopreserved PBSC transplants showed that the procedure is feasible and safe, with high stem cell viability and low rates of complications.* -
  • The study found that stem cell viability was over 90% for multiple myeloma and over 75% for lymphomas, with quick engraftment times, and only 1% transplant-related mortality within 100 days.*
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  • * Both types of HCT are risky and complicated, but global collaboration among medical professionals has established it as a standard treatment for many serious blood-related illnesses, resulting in improved patient outcomes.
  • * Over 1.5 million HCT procedures were performed from 1957 to 2019, and this number continues to rise globally, supported by registries and international medical societies.
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Promoting access to and excellence in hematopoietic cell transplantation (HCT) by collecting and disseminating data on global HCT activities is one of the principal activities of the Worldwide Network for Blood and Marrow Transplantation, a non-governmental organization in working relations with the World Health Organization. HCT activities are recorded annually by member societies, national registries and individual centers including indication, donor type (allogeneic/autologous), donor match and stem cell source (bone marrow/peripheral blood stem cells/cord blood). In 2018, 1,768 HCT teams in 89 countries (6 World Health Organization regions) reported 93,105 (48,680 autologous and 44,425 allogeneic) HCT.

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  • Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy utilizing CRISPR-Cas9 gene editing to increase fetal hemoglobin production in patients with sickle cell disease.
  • A phase 3 study involved 44 patients aged 12 to 35 with a history of severe vaso-occlusive crises; patients received edited stem cells after myeloablative conditioning.
  • Results showed 97% of patients were free from vaso-occlusive crises and 100% avoided hospitalization for over 12 months, with a safety profile similar to standard treatments.
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Objectives: Based on a previous phase 1 study, total marrow irradiation (TMI) at 9Gy was added to a myeloablative FluBu4 conditioning regimen in allogeneic hematopoietic stem cell transplantation (HSCT) for myeloid malignancies. Here, we report on the long-term toxicity of TMI combined with FluBu4 and compare it to patients who received only FluBu4.

Methods: We retrospectively analyzed 38 consecutive patients conditioned with FluBu4/TMI (n = 15) or FluBu4 (n = 23, control group) who had at least 1 year follow-up post-transplant.

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Objective: To perform a longitudinal study for determining the development of ocular graft-versus-host disease (oGVHD) after allogeneic hematopoietic stem cell transplant (HSCT) and report cases that illustrate the "window of opportunity" concept in oGVHD treatment.

Methods: Patients (n=61) were examined at prescheduled clinic visits before HSCT and three-month intervals after HSCT for 2 years. The presence or absence of oGVHD was determined using the international chronic oGVHD consensus group diagnostic criteria.

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We provide a summary of the 4th ASTCT International Workshop with presentations from experts from Chile ("Setting Up a Transplantation Program in Chile," by Dr Pablo Ramirez), Saudi Arabia ("Developing Quality Programs in North Africa," by Dr Amal Alseraihy), and Japan ("The Japanese Transplant Registry Unified Management Program [TRUMP]: Current Issues and the Future," by Dr Yoshiko Atsuta). Workshop objectives included: (1) recognizing the benefits and importance for low- and middle-income countries of developing quality criteria and programs beyond existing accreditation programs, such as the Foundation for the Accreditation of Cellular Therapy (FACT) and the Joint Accreditation Committee ISCT-Europe and EBMT (JACIE); (2) describing the relationships among monitoring outcomes, including mortality, improvement of care, data reporting, and associated costs; and (3) reviewing how quality structures have been implemented and are improving care worldwide.

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Splenomegaly is a hallmark of myelofibrosis, a debilitating haematological malignancy for which the only curative option is allogeneic haematopoietic cell transplantation (HCT). Considerable splenic enlargement might be associated with a higher risk of delayed engraftment and graft failure, increased non-relapse mortality, and worse overall survival after HCT as compared with patients without significantly enlarged splenomegaly. Currently, there are no standardised guidelines to assist transplantation physicians in deciding optimal management of splenomegaly before HCT.

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Purpose: Total marrow irradiation (TMI) involves optimization of extremely large target volumes and requires extensive clinical experience and time for both treatment planning and delivery. Although volumetric modulated arc therapy (VMAT) achieves substantial reduction in treatment delivery time, planning process still presents a challenge due to use of multiple isocenters and multiple overlapping arcs. We developed and evaluated a knowledge-based planning (KBP) model for VMAT-TMI to address these clinical challenges.

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Background: Chemokine receptor CXCR4 antagonist plerixafor (Px) as well as high volume (HV) leukapheresis have been shown to reduce hematopoietic stem progenitor cell (HSPC) mobilization failure rates. However, no direct comparisons of such methods currently exists.

Methods And Materials: We compared the HSPC collection yield based on basal peripheral blood CD34+ cell numbers in patients diagnosed with multiple myeloma or non-Hodgkin's lymphoma undergoing autologous stem cell transplantation in a retrospective chart review.

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Background: Multiple myeloma (MM) predominantly affects older patients; many of whom do not undergo autologous hematopoietic stem cell transplant (AHSCT) despite the associated survival benefits. This study was conceived to investigate the patterns of AHSCT among MM patients with due regard to their age and standardized fitness assessments.

Methods: Fitness scores as per the hematopoietic stem cell transplant-comorbidity index (HSCT-CI) and risk scores as per the revised-myeloma comorbidity index (R-MCI) of MM patients treated between January 2017 and December 2019 were analyzed to assess fitness for AHSCT.

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Blood and marrow transplantation (BMT) is rarely available in many low- to middle-income countries (LMICs). In 2012, Civil Service Hospital, a government hospital in Kathmandu, partnered with the University of Illinois at Chicago to consult on the establishment of BMT in their hospital, train staff, and promote educational activities. The implementation of BMT occurred in 3 phases over 4 years and included regular onsite visits, training of personnel in Chicago, continuous remote communication, and co-organization of educational events in Kathmandu.

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The goal of therapy for patients with essential thrombocythemia (ET) and polycythemia vera (PV) is to reduce thrombotic events by normalizing blood counts. Hydroxyurea (HU) and interferon-α (IFN-α) are the most frequently used cytoreductive options for patients with ET and PV at high risk for vascular complications. Myeloproliferative Disorders Research Consortium 112 was an investigator-initiated, phase 3 trial comparing HU to pegylated IFN-α (PEG) in treatment-naïve, high-risk patients with ET/PV.

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Background: Sickle cell disease (SCD) is a life-limiting inherited hemoglobinopathy that results in significant complications and affects quality of life. Hematopoietic stem cell transplantation (HSCT) is currently the only curative intervention for SCD; however, guidelines are needed to inform how to apply HSCT in clinical practice.

Objective: These evidence-based guidelines of the American Society of Hematology (ASH) are intended to support patients, clinicians, and health professionals in their decisions about HSCT for SCD.

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Background: Whether race/ethnicity plays a role in hematopoietic stem/progenitor cells (HSPC) mobilization in autologous donors has not been studied. We hypothesize that donor characteristic including race/ethnicity, age, sex, body mass index, and diagnostic groups influences HSPC mobilization. Diagnostic groups include healthy allogeneic donors, autologous multiple myeloma (MM) and non-MM donors.

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Establishing a hematopoietic cell transplantation (HCT) program is complex. Planning is essential while establishing such a program to overcome the expected challenges. Authorities involved in HCT program establishment will need to coordinate the efforts between the different departments required to start up the program.

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Non-myeloablative haematopoietic progenitor cell transplantation (HPCT) from matched related donors (MRD) has been increasingly utilized in sickle cell disease (SCD). A total of 122 patients received 300 cGy of total body irradiation (TBI), alemtuzumab, unmanipulated filgrastim-mobilized peripheral blood HPC and sirolimus. The median follow-up was four years; median age at HPCT was 29 years.

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Article Synopsis
  • Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are serious genetic disorders that require ongoing medical treatment and can be life-threatening.
  • Researchers utilized CRISPR-Cas9 technology to modify CD34+ stem cells from healthy donors, targeting a specific enhancer to increase fetal hemoglobin production by altering BCL11A, a gene that suppresses it.
  • Two patients, one with TDT and the other with SCD, received these edited cells after a preparative procedure and showed significant improvements after a year, including high fetal hemoglobin levels, independence from blood transfusions, and reduced complications from SCD.
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National lockdown to control the spread of COVID-19 in Nepal started in March 2020. This lockdown mandated closure of private and public transportation. The patients with hematological malignancies were at risk of delayed consultation, admission and missing scheduled chemotherapy.

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Synopsis of recent research by authors named "Damiano Rondelli"

  • - Damiano Rondelli's recent research focuses on enhancing the practices and understanding of hematopoietic stem cell transplantation (HSCT), particularly in resource-limited settings and emerging countries within the Eastern Mediterranean Region. He emphasizes the importance of knowledge sharing and education among healthcare teams during workshops, aiming to expand HSCT capabilities in these regions.
  • - His publications highlight significant advances in HSCT methodologies, such as non-cryopreserved autologous peripheral blood stem cell transplantation and the role of registries in improving treatment outcomes, aiming to facilitate better access to care and patient safety measures.
  • - Rondelli is also involved in evaluating novel therapies, including CRISPR-Cas9 gene editing for sickle cell disease, and assessing the long-term effects of various conditioning regimens in allogeneic HSCT. His work demonstrates a commitment to optimizing HSCT practices while engaging international collaborations to bridge gaps in transplant access and quality.*