Hematopoietic Stem Cell Transplantation Program Research Articles

Optimization of a home hospitalization program for hematopoietic stem cell transplantation with ehealth integration and clinical pharmacist involvement.

Home hospitalization represents an alternative to traditional hospitalization, providing comparable clinical safety for hematological patients. At-home therapies can range from the delivery of intravenous antibiotics to more complex scenarios, such as the care during the early period after hematopoietic stem cell transplantation and chimeric antigen receptor T-cell therapy. Early discharge from conventional hospitalization is feasible and helps reduce hospital resources and waiting lists. The coordinated efforts of multidisciplinary teams, including hematologists, nurses, and pharmacists, ensure patient safety and continuity of care. The traditional model of home hospitalization relies on home visits and telephone consultations with physicians and nurses. However, the use of eHealth technologies, such as MY-Medula, can enhance communication and monitoring, and thereby improve patient outcomes with no additional costs. The active involvement of a clinical pharmacist in home hospitalization programs is essential, not only for the proper logistical management of the medication but also to ensure its appropriateness, optimize treatment, address queries from the team and patients, and promote adherence. In conclusion, the implementation of hematopoietic stem cell transplantation and chimeric antigen receptor T-cell therapy home hospitalization programs that use both an eHealth tool and a multidisciplinary care model can optimize patient care and improve quality of life without increasing healthcare costs.

Breaking barriers: supporting hematopoietic stem cell transplant program through collaborative radiation therapy service from a physically distant center

BackgroundTotal body irradiation (TBI) for hematopoietic stem cell transplant (HSCT) has certain distinct advantages, such as uniform dose distribution and lack of drug resistance, but it is not widely available in resource-constrained settings. To overcome the limitations of in-house radiotherapy services in hematology centers, we evaluated the feasibility of conducting HSCT programs in coordination with two physically distant centers using a reduced-intensity TBI protocol.MethodsThirty-two patients with a median age of 20.5 years were included in the study. Fifteen patients were diagnosed with aplastic anemia, 10 patients with acute myeloid leukemia (AML), 3 patients with acute lymphocytic leukemia (ALL), and 4 patients with other hematological conditions. Conditioning regimens used were fludarabine plus cyclophosphamide in 29 cases, fludarabine-cytarabine ATG in 2 cases, and busulfan plus fludarabine in 1 case. The TBI dose was 3 Gy in 28 cases and 2 Gy in 4 cases. Patients were followed monthly after TBI, and the major toxicities were recorded.ResultsThe median follow-up was 22 months. The most common acute complication was acute graft-versus-host disease (GVHD), which occurred in 15.6% of patients. The major late complications were chronic GVHD (9.3%), Cytomegalovirus (CMV) infection (34.3%), and CMV-induced secondary graft failure (6.2%). Seventy-five percent of patients were alive, 21.9% were dead, and 1 patient was lost to follow-up.ConclusionsHSCT based on TBI is feasible even if the center lacks a radiotherapy facility by coordinating with a remote radiotherapy facility. without compromising the patient's outcome.

Special Report: Summary of the eighth workshop of the worldwide network for blood and marrow transplantation on the status and issues related to hematopoietic stem cell transplantation in near-east countries, held in Pakistan from September 22 to 23, 2022.

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. International support, collaboration, and local engagement, including government participation and WHO assistance, are valuable in increasing HSCT access worldwide.

Survival of Hematopoietic Stem Cell Transplantation in the National Institute of Neoplastic Diseases, Lima-Perú between 2012 to 2022: 10 Years of Experience

Introduction: Hematopoietic stem cell transplantation (HSCT) is part of standard treatment in a great number of hematologic malignancies and some solid tumors. The National Institute of Neoplastic Diseases (INEN) started the HSCT program in 2012, performing over 600 HSCT so far. In this study, we report the overall survival (OS) at 5 years of patients who undergone this procedure in our institution through 10 years of experience. Methods: We analyzed retrospectively the survival of all patients admitted to the HCST program at INEN between 2012 and 2022. Clinical and pathological data were collected from the medical records and the unit's transplant patient registry. The OS was determined through the Kaplan-Meier method and the comparisons of survival curves were performed using the log-rank or Breslow test. Results: During the study period, a total of 647 transplants were performed. The median age was 39 years (range: 1 - 70), 73.4% of patients were between 18 and 60 years, and 57.1% were men. Most transplants (n = 591, 91.3%) were performed in the adult population and 8.6% (n = 56) in pediatric population. According to the type of transplant, 68% (n=440) were autologous and 32% (n=207) allogeneic from related donor. The most frequent diagnosis were acute leukemia (n = 221, 34.2%), multiple myeloma (n = 190, 29.3%), non-Hodgkin lymphoma (n = 105, 16,2%; of these, 22 were T- cell lymphomas), Hodgkin lymphoma (n = 69, 10.7%) and germinal cell tumor (n = 33, 5.1%); in the pediatric population, leukemia was the most common diagnosis (66.1%). The median follow-up of the patients was 3.7 years (95% CI: 3.4, 3.9), 25.2% (n = 163) of the patients died due to disease progression. The median overall survival (OS) for the entire cohort was not reached and the OS rate at 5 years was 68.3%. The 5-year OS rate by transplant subtype was 53.9% and 75.3% (p < 0.001) for allogeneic and autologous transplants, respectively; and according to the pathology, the patients who had a better 5-year OS were HL (87.5%) and NHL (77.5%), while in leukemias it was 56.1%. Survival by age group (<40 vs >40) and sex (female vs. male) did not present a significant difference (p > 0.05). Conclusions: The survival of the transplanted patients was similar to that reported in other series. Leukemia was the most frequent hematological malignancy admitted to the HSCT program. HL and NHL were the malignant tumors with the highest survival rates at 5 years. Nearly a third of transplants were allogeneic, all from matched related donors. HSCT in our series has shown an improvement in OS at 5 years in both autologous and allogeneic transplant patients compared to non-transplanted patients, according to international reports.

Forging international collaboration and alliances to establish the largest transplant center in the north of Vietnam

Through collaboration with international experts, our institution established a highly active and successful hematopoietic stem cell transplant program, providing access to this potentially curative treatment modality for patients with a variety of benign and malignant hematological diseases. The initial development of an autologous stem cell transplant program provided our institution with the infrastructure, equipment, and expertise needed for the subsequent development of an allogeneic stem cell transplant program. Key transplant staff received training from international transplant experts at the NHLBI/NIH, the Mayo Clinic, the Johns Hopkins Hospital, and Nagoya Japan, providing them with the expertise to conduct a variety of different transplant approaches, including PBSC transplants from HLA-matched relatives, unrelated cord blood transplants, haploidentical transplants, and CD34 selected stem cell transplants. Patient characteristics were varied among all groups. The number of allogeneic and autologous transplants performed at the NIHBT has increased steadily every year since the initiation of our transplant program. By 2022, 547 transplant procedures had been performed, including 268 autologous and 279 allogeneic transplants. Allogeneic transplants were performed for both malignant and nonmalignant hematological diseases, with acute leukemia (AL) being the most common indication for allogeneic HCT. The majority of recipients undergoing allogeneic transplantation received G-CSF mobilized PBSC allografts from either HLA identical or haplo-identical relatives, with a smaller percentage of patients receiving a UCB transplant or a PBSC allograft that had been CD34+ selected. Amongst the 279 recipients of an allogeneic transplant, mortality rates within day 100 and beyond day 100 were 12.6% and 26.2% respectively. Overall survival (OS) and event-free survival at 5 years in benign and malignant subgroups were 81% and 73% vs 52% and 48% respectively. Through collaboration with international transplant experts, the National Institute of Hematology and Blood Transfusion in Hanoi has stood up the most active transplant center in the northern region of Vietnam. Patients coming from low-income financial backgrounds are now able to receive a variety of different state-of-the-art transplant approaches that are affordable and have been associated with excellent long-term outcomes.

Cytopenia: a report of haplo-cord transplantation in twin brothers caused by a novel germline GATA1 mutation and family survey.

Cytopenia due to the abnormal regulation of GATA1 could manifest as varying degrees of thrombocytopenia and/or anemia and more severely in male children than in female children. Here, we describe the case of pancytopenic and transfusion-dependent twin brothers at our center whose bone marrow puncture revealed low bone marrow hyperplasia. Whole-exome sequencing revealed that the twins had a new germline GATA1 mutation (nm_002049: exon 3:c.515 T >C:p.F172S), which confirmed the diagnosis of GATA1 mutation-related pancytopenia. The mutation was inherited from their mother, who was heterozygous for the mutation. Sanger sequencing verified the pathogenicity of the mutation. Further family morbidity survey confirmed that GATA1 mutation-related pancytopenia is an X-linked recessive genetic disorder. We developed haploid hematopoietic stem cell transplantation programs for twins, with the father as the only donor, and finally, the hematopoietic reconstruction was successful. Although they experienced acute graft-versus-host disease, hemorrhagic cystitis, and a viral infection in the early stage, no abnormal manifestations or transplant-related complications were observed 3months after transplantation. Through hematopoietic stem cell transplantation technology for one donor and two receptors, we eventually cured the twins. The p.F172S variant in the new germline GATA1 mutation may play an essential role in the pathogenesis of GATA1 mutation-related cytopenia.

Current use and outcomes of hematopoietic stem cell transplantation: Brazilian summary slides - 2023

The first hematopoietic stem cell transplantation (HSCT) program in Latin America started in 1979 at the federal university hospital in Curitiba, Paraná, Brazil. Over the years, the number of centers performing transplants in our country has increased tremendously, generating the need to know the results of this treatment modality. Understanding the HSCT scenario in Brazil is still challenging, since not all Brazilian centers report data to the Center for International Blood and Marrow Research (CIBMTR). Despite the improvement in the number of reporting centers over the past few years, infrastructure and trained data managers are still lacking. The partnership between the Brazilian Cellular Therapy and Bone Marrow Transplant Society (SBTMO) and the CIBMTR enabled the establishment of the Hematopoietic Stem Cell Transplantation Brazilian Registry (HSCTBR), using the CIBMTR Data Back to Center (DBtC) tool to retrieve Brazilian HSCT data in a standardized and organized way. Since then, it has been possible to gather country-level data on HSCT demographics and transplant outcomes. Between 2012 and 2022, complete information on 9,868 transplants were reported to the CIBMTR from 40 Brazilian transplant centers. The consolidation of the HSCTBR using CIBMTR infrastructure allowed the development and regular update of the Brazilian Summary Slides. Despite the differences in the number of cases and follow-up time, the results in this study were similar to those presented in the United States (US) Summary Slides. In this paper we present the 2023 SBTMO-CIBMTR Summary Slides prepared by the SBTMO data managers (GD-SBTMO).

Outcome of SARS-CoV2 infection in hematopoietic stem cell transplant recipients for autoimmune diseases

Hematopoietic stem cell transplant (HSCT) recipients may be at high risk of mortality from coronavirus disease 2019 (COVID-19). However, specific data on COVID-19 after treatment with HSCT in patients affected by autoimmune diseases (ADs) are still lacking.In this multicenter observational study of the European Society for Blood and Marrow Transplantation (EBMT), clinical data on COVID-19 in 11 patients affected by severe ADs treated with HSCT (n = 3 allogeneic transplant; n = 8 autologous transplant) are reported. All patients were symptomatic during the initial phase of the SARS-CoV-2 infection. At screening, 5 patients reported upper respiratory symptoms, 3 patients had cough without oxygen requirement, and 6 patients exhibited extra-pulmonary symptoms. Four cases developed a lower respiratory tract disease (LRTD). Hospitalization was required in 6 cases, without necessity of intensive care unit (ICU) admission and/or ventilation/supplemental oxygen. Different interventions were adopted: remdesivir (n = 1), nirmatrelvir/ritonavir (n = 1), sotrovimab (n = 1), immunoglobulins (n = 1). At last follow-up, all patients are alive and had resolution of the infection.The current analysis describing the mild-moderate course of COVID-19 in transplant recipients affected by ADs, similar to the course observed in ADs under standard treatments, provides useful information to support the delivery of HSCT programs in this field. Vaccination and new treatments available for SARS-CoV-2 may be useful to further minimize the risk of infection.

398 - Help for Ukrainian Hematology Patients (HUP): A Global Initiative Supporting Hematopoietic Stem Cell Transplantation (HSCT) Programs in a Time of Conflict

398 - Help for Ukrainian Hematology Patients (HUP): A Global Initiative Supporting Hematopoietic Stem Cell Transplantation (HSCT) Programs in a Time of Conflict

A Hematopoietic Stem Cell Transplantation Startup in Iraqi Kurdistan: Results in Thalassemia Patients and Analysis of the Methodology

In hemoglobinopathy-prone regions, like the Middle East, thalassemia is the most prevalent noncommunicable life-threatening disorder of children and is highly curable by hematopoietic stem cell transplantation (HSCT). Moreover, transplantation is very cost-effective, and HSCT programs can be established directly in middle-income countries (MICs) at a reduced cost while maintaining quality standards and outcomes consistent with international ones. The aim of the present study was to review and verify the efficacy of the applied methodology through the analysis of 47 consecutive matched-related HSCTs in children with thalassemia. In 2016, the first HSCT unit for adults and children with both malignant and nonmalignant diseases was developed in Iraqi Kurdistan, thanks to a capacity building project funded by the Italian Agency for Development Cooperation. Data on clinical activity were obtained from a cohort of patients treated in the newly established HSCT unit. Primary endpoints were overall survival (OS) and thalassemia-free survival (TFS). Startup of the HSCT unit was completed over a 3-year period. Assessing and meeting minimum requirements were crucial for the startup; moreover, a team of international health care professionals (HCPs), all experts in the field of HSCT, conducted the education and training phase, involving all the clinical and nonclinical professionals in the program. At a median follow-up of 2.6 years, the 3-year TFS and OS were 82.8% (SE, 5.5%) and 87.1% (SE, 4.9%), respectively. TFS and graft-versus-host-disease-free composite survival was 80.6% (SE, 5.8%). At present, the HSCT service is completely autonomous, and more than 250 transplants have been done in both adults and children. The minimal essential requirements for an HSCT startup may be affordable in many MICs. Our results for thalassemia are comparable with international data. A twinning program with an international group of experts and a capacity-building approach is crucial for the success of the program, a strategy that allows for rapid development of HSCT units.

Pediatric Hematopoietic Stem Cell Transplantation in Lithuania - 20 Years of Progress through Collaboration.

Hematopoietic stem cell transplantation (HSCT) is a complex procedure that is curative for several fatal pediatric malignancies and non-malignant diseases. Despite its complexity, potential toxicity, and high costs HSCT has become a standard procedure worldwide for several decades. Pediatric HSCT programs encounter several specific challenges. The rarity and heterogeneity of primary diseases, result in an almost 10-fold inferior number of pediatric HSCT as compared to adults. In contrast to the adult programs, where autologous HSCT is more common, allogeneic HSCT (that is more complex) prevails in pediatric setting which is underpinned by a higher number of inborn disorders transplanted in early childhood.In Lithuania, the pediatric HSCT program (EBMT CIC* 508) was launched at Vilnius University Hospital Santaros Klinikos in February 2002. Currently, this is the only specialized pediatric HSCT center in Lithuania and in the Baltic countries. Since 2011 it is a reference center for Latvian children who need autologous or allogeneic transplantation.Here we summarize conference proceedings presented at the scientific event “Pediatric hematopoietic stem cell transplantation in Lithuania – 20 years of progress through collaboration”. The meeting held on September 22-23, 2022, in Vilnius and aimed at commemorating 20 years of the launch of the pediatric transplant program in Lithuania. The event pursued sharing the experience in the field of pediatric HSCT in the Baltic countries. Given a very small population in all three Baltic countries, Lithuania, Latvia, and Estonia face an additional challenge in maintaining sufficient transplant volume and gaining experience. Several distinguished speakers from USA, Denmark, Italy, Germany, Spain, UK and Ukraine shared their expertise in the field and emphasized the crucial role of national and international collaboration to achieve progress in the management of this very rare and complex procedure that offers cure for otherwise fatal pediatric conditions.

ABCL-070 Multicenter Randomized Controlled (Comparative) Open Prospective Study to Evaluate the Efficacy of the R-DA-EPOCH-21 and R-mNHL-BFM-90 ± Autologous Hematopoietic Stem Cell Transplantation Programs in Untreated Patients With Diffuse B-Cell Large Cell Lymphoma With Signs of Poor Prognosis - DLBCL-2015 Protocol

Short-pulse NHL-BFM-90 chemotherapy is highly effective in pediatric aggressive B-cell lymphomas. We modified R3 arm of NHL-BFM-90 program (R-mNHL-BFM-90) for adults under 60 years with high-risk de novo DLBCL and conducted the randomized study compared with R-DA-EPOCH-21 chemotherapy. To evaluate efficacy and toxicity in de novo adult DLBCL patients with 2 or more signs of poor prognosis. Treatment protocol was registered as NCT02842931 at www. gov. Inclusion criteria were newly diagnosed DLBCL, not otherwise specified (NOS), according to WHO, 2008 and 2017 criteria, intermediate and high age-adjusted International Prognostic Index (aaIPI) risk, age 18-60. Response was corresponded by Lugano 2014 criteria. The study design intended randomization for four parallel arms: -6-cycle R-DA-EPOCH-21; - 6-cycle R-DA-EPOCH-21 plus auto-HSCT; - 6-cycle (A-B-A-B-A-B) R-mNHL-BFM-90; - 6-cycle R-mNHL-BFM-90 plus auto-HSCT. If CR wasn't achieved after treatment in every arm additionally 2-cycle R-DHAP chemotherapy were completed before auto-HSCT. A total of 140 patients from 13 Russian clinics were included from February 2015 to June 2021. Randomization was done according "intention-to-treat" as follows: R-DA-EPOCH-21 (35 patients); R-DA-EPOCH-21+auto-HSCT (30 patients); R-mNHL-BFM-90 (37 patients); R-mNHL-BFM-90+auto-HSCT (38 patients). First-step analysis was carried out for 130 patients according "intention-to-treat." In all cases, the final diagnosis was assessed as indolent lymphoma transformation into DLBCL or other "non-DLBCL" lymphomas. Second-step analysis was performed for 116 "proved" de novo DLBCL patients. Evaluation of comparative efficacy was carried out in R-DA-EPOCH-21±auto-HSCT and R-mNHL-BFM-90±auto-HSCT cohorts. Hematological/non-hematological toxicity was acceptable in all groups. However, neutropenic fever and grade 3-4 thrombocytopenia differed statistically significantly (χ2 test, p<0.05) between R-DA-EPOCH-21 and R-mNHL-BFM-90 arms. The value of auto-HSCT will be determined later. De novo DLBCL NOS is potentially a curable disease with no relapses. R-mNHL-BFM-90 program is highly effective in de novo DLBCL NOS adult patients. R-mNHL-BFM-90 therapy toxicity is acceptable. The estimated CR duration probability within 10 months after treatment completion in the high-risk group is 100% in R-mNHL-BFM-90 group compared to 57% in R-DA-EPOCH group (χ2 test, p=0.0047).

A hub-and-spoke model to deliver effective access to chimeric antigen receptor T-cell therapy in a public health network: the Catalan Blood and Tissue Bank experience

Background aimsTo describe and analyze whether a hub-and-spoke organizational model could efficiently provide access to chimeric antigen receptor (CAR) T-cell therapy within a network of academic hospitals and address the growing demands of this complex and specialized activity. MethodsThe authors performed a retrospective evaluation of activity within the Catalan Blood and Tissue Bank network, which was established for hematopoietic stem cell transplantation to serve six CAR T-cell programs in academic hospitals of the Catalan Health Service. Procedures at six hospitals were followed from 2016 to 2021. Collection shipments of starting materials, CAR T-cell returns for storage and infusions for either clinical trials or commercial use were evaluated. ResultsA total of 348 leukocytapheresis procedures were performed, 39% of which were delivered fresh and 61% of which were cryopreserved. The network was linked to seven advanced therapy medicinal product manufacturers. After production, 313 CAR T-cell products were shipped back to the central cryogenic medicine warehouse located in the hub. Of the units received, 90% were eventually administered to patients. A total of 281 patients were treated during this period, 45% in clinical trials and the rest with commercially available CAR T-cell therapies. ConclusionsA hub-and-spoke organizational model based on an existing hematopoietic stem cell transplantation program is efficient in incorporating CAR T-cell therapy into a public health hospital network. Rapid access and support of growing activity enabled 281 patients to receive CAR T cells during the study period.

Conducting hematopoietic stem cell transplantation in low and middle income countries

ABSTRACT Background Hematopoietic stem cell transplantation (HSCT) is a well-recognized therapeutic procedure; costs limit its widespread use in low and middle income countries (LMIC). Methods Over a 30-year period, we have conducted HSCT in LMIC, making adaptations to the conventional procedures conducted in high-income countries (HIC). Results These salient observations stem from our practice: (1) Start with autologous transplantations in patients with hematological malignancies, specifically multiple myeloma; cell freezing devices are not necessary. (2) Next, consider auto-HSCT in patients with autoimmune diseases. (3) Introduce allogeneic transplants, initially using reduced intensity conditioning regimens. Conducting the HSCT on an outpatient basis is cheaper and safer. (4) Do not build HEPA-filtered rooms nor laminar flow cabins. (5) Do not graft cord blood cells nor start a cord blood blank. (6) Engage in haploidentical transplantations which are more feasible and cost-effective. (7) Matched unrelated donors are extremely expensive. (8) Use generic drugs and biosimilars. (9) Blood product irradiation devices are not necessary. (10) Do not try to reproduce other HSCT programs from HIC; develop your own methods. Conclusions HSCT can be conducted in LMIC with reduced costs and similar efficacy, thus making this therapeutic option affordable for more persons.

BK virus kao uzročnik hemoragičnog cistitisa kod bolesnika lečenih alogenom transplantacijom koštane srži

Introduction: BK polyomavirus is a double-stranded DNA virus from the Polyomaviridae family. According to DNA sequences, this virus can be classified into six genotypes. In hematological patients enrolled in allogeneic hematopoietic stem cell transplantation (HSCT) programs, it can lead to hemorrhagic cystitis. Aim: The aim of this study is calculating the prevalence of BK polyomavirus PCR (polymerase chain reaction) positivity in the blood and urine of patients involved in allogeneic HSCT, determining the predictive factors for clinical presentation of BK polyomavirus-associated hemorrhagic cystitis, as well as determining its effects on overall survival (OS) of the patients. Materials and methods: This retrospective cohort study enrolled 42 patients from the Clinic of Hematology of the University Clinical Center of Serbia. The presence of the virus in blood and urine was determined by the PCR method. The survival rate of the patients in relation to hemorrhagic cystitis was calculated with the Kaplan-Meier method and comparison was performed with the log-rank test. Results: A positive PCR result in the blood was found in 97.6% of the subjects, while urine tested positive in 100% of patients. The estimated survival time in patients without hemorrhagic cystitis was 44.357 months, while the group with the clinical presentation of hemorrhagic cystitis had an estimated survival time of 17.395 months. Based on the log-rank test, we found a significant difference in survival between those groups of patients (p = 0.049). With regards to leukocyte engraftment day, patients engrafted after D+14, had a higher frequency of hemorrhagic cystitis (p = 0.037). Conclusion: BK polyomavirus-associated hemorrhagic cystitis is a common complication of treatment in patients suffering from hematological malignancies who are enrolled in an alo-HSCT program, and has a significant impact on OS..

Extracorporeal photopheresis in chronic graft-versus-host disease: clinical description and economic study

IntroductionAlthough extracorporeal photopheresis (ECP) is a promising second-line therapy in the treatment of chronic graft-versus-host disease (cGVHD), its use is limited by its high cost. This study aims to describe the clinical evolution of patients who underwent ECP therapy for cGVHD and to perform an economic analysis of the therapy MethodsThis was a case series between 2016 and 2020 describing the clinical response to ECP and a micro-cost analysis of the therapy using time-driven activity-based costing. ResultsSix patients underwent ECP for corticosteroid-dependent cGVHD The cost per ECP session is 14,960.90 Brazilian reais (BRL), which primarily consists of the ECP kit with an activator (82.78%), followed by the hospital's physical structure (14.66%), human resources (2.48%) and exams/inputs (0.08%). The number of sessions performed ranged from 2 to 42. The total cost of the therapy per patient ranged from BRL 30,000 to 500,000. ConclusionThe response of the patient with cGVHD to treatment with ECP was variable. These micro-costing results can be used to develop remuneration and cost control strategies in hematopoietic stem cell transplantation programs, as well as in further economic studies.

Helicobacter pylori antibodies and multiple sclerosis: a single-center study and a short review of the literature

BackgroundMultiple sclerosis is an immune-mediated disease which has been associated to a great variety of mechanisms that could influence its pathogenesis. Numerous reports in the medical literature suggest that Helicobacter pylori may be a mediator of the disease. However, it is unknown if there is any clear association between MS and HP.ResultsWe studied 144 persons with multiple sclerosis prospectively enrolled in our hematopoietic stem cell transplantation program. In 144 persons, 14% patients were positive for IgG-HP whereas 86% were negative, 8.3% pwMS were IgM-HP positive while 91.6% pwMS were negative, 18% patients were positive and 82% negative for IgA-HP. Significantly lower concentrations of anti-HP IgG were found in RRMS in comparison with SPMS (− 28.5, 95% CI 4.3–52.7). While concentrations of anti-HP IgA were significantly lower in SPMS in comparison with RRMS (0.54, 95% CI 0.1–0.9). In a multivariate analysis, positivity rate of anti-HP IgG was found to be higher in SPMS patients (OR 4.7, 95% CI 1.1–19.6).ConclusionsThere was a negative correlation between the presence of anti-HP antibodies and MS. Further larger studies with specific laboratory testing methods are needed to discard or confirm the potential role of anti-HP antibodies as protective for MS.

Multidisciplinary Development Issues of Hematopoietic Stem Cell Transplantation Program in Ukraine: Role of Auxiliary Cryopreservation Technologies

Hematopoietic stem cell transplantation (HSCT) is a life-saving medical technology for many serious diseases. Active international exchange of transplant material is ensured through productive cooperation of world international donation, transplantation, cell therapy organizations, along with their associations. Analysis of the experience of many countries has allowed the development of key recommendations from the Worldwide Network for Blood and Marrow Transplantation for establishing HSCT programs. According to them, to make the most effective use of the capabilities of this medical technology, the creation of new transplant programs requires both sufficient investment and the presence of specialized professional teams for multidisciplinary support of the entire process.&#x0D; This article discusses prospects for the development of the national transplant program in Ukraine. In particular, the role of Ukrainian national scientific and practical traditions detailed in the creation of cellular processing technologies and cryopreservation as part of the team support providing components of transplantation medical technology. It is looked forward that the development of the HCST program in Ukraine will take place through continuous improvement in order to meet the criteria of the highest quality and safety. Its serious basis is the solid scientific traditions, historical and modern experience of many directions that provide the field.

Treosulfan-induced myalgia in pediatric hematopoietic stem cell transplantation identified by an electronic health record text mining tool

Treosulfan is increasingly used as myeloablative agent in conditioning regimen prior to allogeneic hematopoietic stem cell transplantation (HSCT). In our pediatric HSCT program, myalgia was regularly observed after treosulfan-based conditioning, which is a relatively unknown side effect. Using a natural language processing and text-mining tool (CDC), we investigated whether treosulfan compared with busulfan was associated with an increased risk of myalgia. Furthermore, among treosulfan users, we studied the characteristics of given treatment of myalgia, and studied prognostic factors for developing myalgia during treosulfan use. Electronic Health Records (EHRs) until 28 days after HSCT were screened using the CDC for myalgia and 22 synonyms. Time to myalgia, location of pain, duration, severity and drug treatment were collected. Pain severity was classified according to the WHO pain relief ladder. Logistic regression was performed to assess prognostic factors. 114 patients received treosulfan and 92 busulfan. Myalgia was reported in 37 patients; 34 patients in the treosulfan group and 3 patients in the busulfan group (p = 0.01). In the treosulfan group, median time to myalgia was 7 days (0–12) and median duration of pain was 19 days (4–73). 44% of patients needed strong acting opiates and adjuvant medicines (e.g. ketamine). Hemoglobinopathy was a significant risk factor, as compared to other underlying diseases (OR 7.16 95% CI 2.09–30.03, p = 0.003). Myalgia appears to be a common adverse effect of treosulfan in pediatric HSCT, especially in hemoglobinopathy. Using the CDC, EHRs were easily screened to detect this previously unknown side effect, proving the effectiveness of the tool. Recognition of treosulfan-induced myalgia is important for adequate pain management strategies and thereby for improving the quality of hospital stay.

Significance of regional population HLA immunogenetic datasets in the efficacy of umbilical cord blood banks and marrow donor registries: a study of Cretan HLA genetic diversity

Background aimsThe high genetic diversity of HLA across populations significantly confines the effectiveness of a donor or umbilical cord blood search for allogeneic hematopoietic stem cell transplantation (HSCT). This study aims to probe the HLA immunogenetic profile of the population of Crete, a Greek region with specific geographic and historical characteristics, and to investigate potential patterns in HLA distribution following comparison with the Deutsche Knochenmarkspenderdatei (DKMS) donor registry. It also aims to highlight the importance of regional public cord blood banks (PCBBs) in fulfilling HSCT needs, especially in countries with significant genetic diversity. MethodsA cohort of 1835 samples representative of the Cretan population was typed for HLA class I (HLA-A, HLA-B, HLA-C) and class II (HLA-DRB1, HLA-DQB1, HLA-DPB1) loci by high-resolution second field next-generation sequencing. Data were compared with the respective HLA profiles of 12 DKMS populations (n = 20 032). Advanced statistical and bioinformatics methods were employed to assess specific intra- and inter-population genetic indexes associated with the regional and geographic distribution of HLA alleles and haplotypes. ResultsA considerable HLA allelic and haplotypic diversity was identified among the Cretan samples and between the latter and the pooled DKMS cohort. Even though the HLA allele and haplotype frequency distribution was similar to regions of close geographic proximity to Crete, a clinal distribution pattern from the northern to southern regions was identified. Significant differences were also observed between Crete and the Greek population of DKMS. ConclusionsThis study provides an in-depth characterization of the HLA immunogenetic profile in Crete and reveals the importance of demographic history in HLA heterogeneity and donor selection. The novel HLA allele and haplotype frequency comparative data between the Cretan and other European populations signify the importance of regional PCBBs in prioritizing HLA diversity to efficiently promote the HSCT program at the national level and beyond.