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1.
Fanconi anemia phenotypic and transplant outcomes' associations in Iranian patients
Ansari, F., Behfar, M., Naji, P., Darvish, Z., Rostami, T., Mohseni, R., Alimoghaddam, K., Salajegheh, P., Ahadi, B., Mardani, M., et al
Health science reports. 2023;6(4):e1180
Abstract
OBJECTIVES Fanconi anemia (FA) is a rare, heterogeneous, inherited disorder. Allogeneic hematopoietic stem cell transplantation (HSCT) represents the only therapeutic option to restore normal hematopoiesis. This study reports the outcomes of FA-HSCT patients and identifies factors, including clinical phenotype. Our team examined more than 95% of Iranian FA patients during the last decade. STUDY DESIGN One hundred and six FA patients (age range: 2-41) who underwent HSCT from March 2007 to February 2018 were enrolled. Clinical characteristics of genetic disease, pre-HSCT findings, HSCT indication, and long-term follow-up evaluated and recorded. Data were analyzed using SPSS 19.0. RESULTS The mean follow-up period for survivors was 36 months (range, 1-101). The 3-year overall survival (OS) and disease-free survival were 72.2% and 71.2%, respectively. The 3-year OS rate for patients with limited and extensive malformations was 78.8% and 56.6%, respectively (p = 0.025). Acute graft versus host disease incidence was 60.52% for patients with limited malformations versus 70% for patients with extensive ones (p = 0.49). Chronic graft versus host disease incidence for these two groups was 9.21% and 10%, respectively (p = 0.91). CONCLUSIONS OS was not associated with each of the malformations singly; however, it was lower in the extensive group. The younger age of patients at the HSCT time leads to a higher OS. The differences in FA patients' outcomes and the various genotypes were probably related. These data provide a powerful tool for further studies on genotype-phenotype association with HSCT results.
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2.
Risk factors for a severe disease course in children with SARS-COV-2 infection following hematopoietic cell transplantation in the pre-Omicron period: a prospective multinational Infectious Disease Working Party from the European Society for Blood and Marrow Transplantation group (EBMT) and the Spanish Group of Hematopoietic Stem Cell Transplantation (GETH) study
Averbuch, D., de la Camara, R., Tridello, G., Knelange, N. S., Bykova, T. A., Ifversen, M., Dobsinska, V., Ayas, M., Hamidieh, A. A., Pichler, H., et al
Bone marrow transplantation. 2023;:1-9
Abstract
Risk factors for severe SARS-Cov-2 infection course are poorly described in children following hematopoietic cell transplantation (HCT). In this international study, we analyzed factors associated with a severe course (intensive care unit (ICU) admission and/or mortality) in post-HCT children. Eighty-nine children (58% male; median age 9 years (min-max 1-18)) who received an allogeneic (85; 96%) or an autologous (4; 4%) HCT were reported from 28 centers (18 countries). Median time from HCT to SARS-Cov-2 infection was 7 months (min-max 0-181). The most common clinical manifestations included fever (37; 42%) and cough (26; 29%); 37 (42%) were asymptomatic. Nine (10%) children following allo-HCT required ICU care. Seven children (8%) following allo-HCT, died at a median of 22 days after SARS-Cov-2 diagnosis. In a univariate analysis, the probability of a severe disease course was higher in allo-HCT children with chronic GVHD, non-malignant disease, immune suppressive treatment (specifically, mycophenolate), moderate immunodeficiency score, low Lansky score, fever, cough, coinfection, pulmonary radiological findings, and high C-reactive protein. In conclusion, SARS-Cov-2 infection in children following HCT was frequently asymptomatic. Despite this, 10% needed ICU admission and 8% died in our cohort. Certain HCT, underlying disease, and SARS-Cov-2 related factors were associated with a severe disease course.
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3.
Report on hematopoietic cell transplantations performed in 2018/2019 focusing on the trends of selection of stem cell sources in the Asia-Pacific region: APBMT Activity Survey
Iida, M., Liu, K., Huang, X. J., Huang, H., Kuwatsuka, Y., Moon, J. H., Lee, J. W., Lakshmi, K. M., Dodds, A., Wilcox, L., et al
Blood cell therapy. 2023;6(4):114-123
Abstract
The number of hematopoietic stem cell transplantations (HCTs) is increasing annually worldwide, and the Asia-Pacific (AP) region is no exception. We report on the absolute number of HCTs in 2018 and 2019 and the trends in graft selection and disease indication in the past few decades. In 2018, 24,292 HCTs were performed in the AP region, of which 8,754 (36.0%) were autologous and 15,538 (64.0%) were allogeneic. Among the allogeneic HCTs, 10,552 (67.9%) of the recipients were related to their donors, whereas 4,986 (32.1%) were unrelated. In 2019, 27,583 HCTs were reported, of which 17,613 (63.9%) were allogeneic and 9,970 (36.1%) were autologous. Although, in 2010, there was a nearly equal number of related and unrelated HCTs, the difference has shown an annual increase, with more than double (2.05) the number of related than unrelated HCTs in 2019. Recent trends in the AP region show that peripheral blood has overwhelmingly surpassed the bone marrow as a graft source for both related and unrelated HCTs, with the haploidentical donor type being preferred; however, their trends in each country/region were quite different among countries/regions. In 2019, the main conditions requiring HCT were acute myelogenous leukemia (n=6,629 [24.0%]), plasma cell disorders (PCD) (n=4,935 [17.9%]), malignant lymphoma (ML) (n=4,106 [14.9%]), acute lymphoblastic leukemia (AML) (n=3,777 [13.7%]), myelodysplastic syndrome or myelodysplastic/myeloproliferative neoplasm (n=1,913 [6.9%]), severe aplastic anemia (n=1,671 [6.1%]), and hemoglobinopathy (n=910 [3.3%]). PCD and ML were the main indications for autologous HCT, and the number of PCD cases has grown more prominent than the corresponding of ML. The increased number of allogeneic transplants for hemoglobinopathy remains prominent, as well as that of AML and acute lymphocytic leukemia for the past 5 years. There was a significant regional variation in the number of facilities performing HCTs, ranging from one in Mongolia and Nepal to 313 in Japan, and differing regional densities varying from 0.1 in Indonesia and Pakistan to 24.7 in Japan. The total transplant density per 10 million population in each country/region also differed (0.2 in Indonesia and 627 in New Zealand). This annual Activity Survey aims to help all participating countries/regions understand the changes in HCT, serve as an asset in promoting HCT activities in the AP region, and be used as a reference for comparison with other registries from Europe and the United States.
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4.
The Effect of Early BCG-Vaccination of SCID Pediatric Patients on the Outcome of Hematopoietic Stem Cell Transplantation Using Reduced Conditioning Regimen
Hamidieh, A. A., Jafari, L., Behfar, M., Karamlou, Y., Shamsipour, M., Mohseni, R., Farajifard, H., Salajegheh, P.
Transplantation and cellular therapy. 2022
Abstract
BACKGROUND The eminence of Bacillus Calmette-Guerin (BCG) vaccine in the vaccination program of newborns, especially in low-income developing countries where tuberculosis is prevalent, has been conspicuous throughout the years; however, the application of the aforementioned vaccine is not without constraints, especially in patients afflicted with immunodeficiency diseases, such as Severe Combined Immunodeficiency (SCID). OBJECTIVE The desideratum of this study is to evaluate whether or not the administration of BCG vaccine upon birth could attenuate the success of hematopoietic stem cell transplantation (HSCT) in SCID pediatric patients STUDY DESIGN In the current study, 30 SCID patients who underwent HSCT using reduced intensity conditioning regimen (RIC) were followed-up for 2 years post-HSCT. The outcome of HSCT was evaluated in both non-BCG-vaccinated (12 patients) and BCG-vaccinated (18 patients) groups. RESULTS Our results demonstrated that patients who had been BCG vaccinated displayed higher incidences of acute GvHD (aGvHD); however, the incidence of chronic GvHD did not differ between the two groups. The overall survival (OS) rate in patients who received the BCG vaccine was similar than their non-vaccinated counterparts. We speculate that the reason that BCG-vaccinated individuals' survival rate, despite having the risk factor of being BGC-vaccinated, was similar to the non-BCG-vaccinated group was due to the fact that they received RIC as their conditioning regimen. CONCLUSION The result of our study indicated that early BCG vaccination was associated with a higher risk of aGVHD. Additionally, the OS of BCG-vaccinated patients was similar in comparison to their non-vaccinated counterparts; alluding to the possibility that the same RIC regimen might be the reason for such outcome, since no other variation between the two groups existed. Regarding to the BCG vaccine affect the outcome of HSCT, we suggest that the administration of BCG vaccine should be deferred until three months of age so that apt testing, without the interference of maternal antibodies, could be performed; however, it is noteworthy that this study could benefit from a bigger cohort in order to further validate the outcome, as the possible reason for some factors not becoming statistically significant was the small sample size of this study.
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5.
Trends in disease indications for hematopoietic stem cell transplantation in the Asia-Pacific region: A report of the Activity Survey 2017 from APBMT
Iida, M., Liu, K., Huang, X. J., Depei, W., Kuwatsuka, Y., Moon, J. H., Dodds, A., Wilcox, L., Ko, B. S., Hamidieh, A. A., et al
Blood cell therapy. 2022;5(4):87-98
Abstract
The Asia-Pacific Blood and Marrow Transplantation Group (APBMT) has been conducting annual surveys on the activity of hematopoietic stem cell transplants since 2007. The APBMT Data Center collected the following data in 2017. A total of 21,504 transplants were registered from 733 transplant centers of 20 countries/regions in the Asia-Pacific (AP) region. Five countries/regions comprised 89.4% of all transplants - China (6,979), Japan (5,794), South Korea (2,626), India (2,034), and Australia (1,789). The number of centers in these five countries/regions also comprised 88.9% of all centers: Japan (373), China (123), India (66), Australia (45), and South Korea (44). The overall ratio between autologous and allogeneic transplants was 37.0% and 63.0%, respectively, but the ratios varied significantly among countries/regions. Autologous transplants have surpassed allogeneic transplants in Thailand, Australia, Vietnam, New Zealand, Singapore, and Iran. In contrast, the proportion of allogeneic transplants comprised over 70% of all transplants in Pakistan, China, and Hong Kong. These ratios were compared by the Data Center among countries/regions that performed more than 50 transplants. The proportion of related and unrelated transplants also differed among countries/regions. The number of unrelated transplants was more than related ones in Japan (2,551 vs. 1,202) and Australia (329 vs. 291), whereas more than 80% of all transplants were related transplants in Malaysia (90.9%), India (89.5%), Iran (87.2%), Vietnam (85.7%), China (80.9%), and Thailand (80.6%). All transplant activities were related transplants in Pakistan, the Philippines, Myanmar, and Nepal, and no allogeneic transplants were performed in Bangladesh and Mongolia. Regarding the indications for transplants, acute myeloid leukemia (AML) was the most common disease for allogeneic transplant (4,759, 35.1% of allogeneic transplants), while plasma cell disorder (PCD) was the most common disease for autologous transplant (3,701, 27.3% of all autologous transplants). Furthermore, the number of transplants for hemoglobinopathy has steeply increased in this region compared with the rest of disease indications (677, 3.1% of all transplants). APBMT covers a broad area globally, including countries/regions with diverse disease distribution, development of HSCT programs, population, and economic power. Consistent and continuous activity surveys considering those elements in each country/region revealed the HSCT field's diverse characteristics and background factors in this region.
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6.
One and a half million hematopoietic stem cell transplants: continuous and differential improvement in worldwide access with the use of non-identical family donors
Niederwieser, D., Baldomero, H., Bazuaye, N., Bupp, C., Chaudhri, N., Corbacioglu, S., Elhaddad, A., Frutos, C., Galeano, S., Hamad, N., et al
Haematologica. 2021
Abstract
The Worldwide Network of Blood and Marrow Transplantation (WBMT) pursues the mission of promoting hematopoietic cell transplantation (HCT) for instance by evaluating activities through member societies, national registries and individual centers. In 2016, 82,718 first HCTs were reported from 1662 HCT teams in 86 of the 195 World Health Organization member states representing a global increase of 6.2% in autologous and 7.0% in allogeneic HCT and bringing the total to 1,298,897 procedures. Assuming a frequency of 84,000/year, 1.5 million HCTs had been performed by 2019 from 1957. Slightly more autologous (53.5%) than allogeneic and more related (53.6%) than unrelated HCTs were reported. A remarkable increase was noted in haploidentical related HCT for leukemias and lymphoproliferative diseases, but even more in non-malignant diseases. Transplant rates (TR; HCT/10 million population) varied according to region reaching 560.8 in North America, 438.5 in Europe, 76.7 in Latin America, 53.6 in South East Asia/Western Pacific (SEA/WPR) and 27.8 in African/East Mediterranean (AFR/EMR). Interestingly, haploidentical TR amounted to 32% in SEA/WPR and 26% in Latin America, but only 14% in Europe and EMR and 4.9% in North America of all allogeneic HCT. HCT team density (teams/10 million population) was highest in Europe (7.7) followed by North America (6.0), SEA/WPR (1.9), Latin America (1.6) and AFR/EMR (0.4). HCTs are increasing steadily worldwide with narrowing gaps between regions and greater increase in allogeneic compared to autologous activity. While related HCT is rising, largely due to increase in haploidentical HCT, unrelated is plateauing and cord blood in decline.
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7.
Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?-A multicenter EBMT-PDWP study
Willasch, A. M., Peters, C., Sedlacek, P., Dalle, J. H., Kitra-Roussou, V., Yesilipek, A., Wachowiak, J., Lankester, A., Prete, A., Hamidieh, A. A., et al
Bone marrow transplantation. 2020
Abstract
Although most children with acute lymphoblastic leukemia (ALL) receive fractionated total body irradiation (FTBI) as myeloablative conditioning (MAC) for allogeneic hematopoietic stem cell transplantation (allo-HSCT), it is an important matter of debate if chemotherapy can effectively replace FTBI. To compare outcomes after FTBI versus chemotherapy-based conditioning (CC), we performed a retrospective EBMT registry study. Children aged 2-18 years after MAC for first allo-HSCT of bone marrow (BM) or peripheral blood stem cells (PBSC) from matched-related (MRD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to control pretreatment imbalances of the observed variables. 3.054 patients were analyzed. CR1 (1.498): median follow-up (FU) after FTBI (1.285) and CC (213) was 6.8 and 6.1 years. Survivals were not significantly different. CR2 (1.556): median FU after FTBI (1.345) and CC (211) was 6.2 years. Outcomes after FTBI were superior as compared with CC with regard to overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and nonrelapse mortality (NRM). However, we must emphasize the preliminary character of the results of this retrospective "real-world-practice" study. These findings will be prospectively assessed in the ALL SCTped 2012 FORUM trial.
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8.
Posterior Reversible Encephalopathy Syndrome after Allogeneic Stem Cell Transplantation in Pediatric Patients with Fanconi Anemia, a Prospective Study
Behfar, M., Babaei, M., Radmard, A. R., Kooraki, S., Farajifard, H., Naji, P., Taebi, S., Hamidieh, A. A.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2020
Abstract
Posterior reversible encephalopathy syndrome (PRES) is one of the most common neurologic complications following hematopoietic stem cell transplantation (HSCT). We aimed to evaluate incidence, clinical, and imaging features of PRES in pediatric patients with FA following HSCT. This prospective study was carried out on all post-HSCT patients with underlying FA disease between 2014 and 2017. Brain CT scan and MRI were performed in all individuals who developed neurologic symptoms. The diagnosis of PRES had done based on clinic-radiological evidence. Follow-up MRI was carried out in all patients with PRES within two months. Forty-one patients with FA (28 males, mean age: 8.19±3.25 years) were enrolled. Out of 15 patients with acute neurologic symptoms, PRES was diagnosed in 9 individuals (21.95%). The occurrence of PRES was significantly higher among patients who had a donor with a one-locus mismatch (p-value: 0.02). Donor relation, stem cell source, and GvHD grading did not have any significant association with the development of PRES. MRI showed asymmetric vasogenic edema in five patients, overt infarct in one, and foci of micro-hemorrhage in three individuals that one of them developing hemorrhagic infarct. The patient expired shortly, while persistent micro-hemorrhage was noted on the other two patients. The result of our study demonstrated the risk of developing PRES after HSCT is higher in FA compared to other diseases reported in articles (21.95% vs. 1-10%), and in contrast to its term, it might be irreversible and has adverse effects on the results of HSCT. Increased vascular and endothelial fragility in FA may impact on the higher frequency of PRES in these individuals.
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9.
Physical therapy pathway and protocol for patients undergoing hematopoietic stem cell transplantation: Recommendations from The Eastern Mediterranean Blood and Marrow Transplantation (EMBMT) Group
Mohammed, J., Aljurf, M., Althumayri, A., Almansour, M., Alghamdi, A., Hamidieh, A. A., ElHaddad, A., Othman, T. B., Bazarbachi, A., Almohareb, F., et al
Hematology/oncology and stem cell therapy. 2019
Abstract
Patients undergoing hematopoietic stem cell transplantation (HSCT) are often referred for physical therapy (PT) to help improve their quality of life. However, to our knowledge there is no clear PT pathway to guide therapists and patients before, during, and after HSCT. This paper not only reviews the current evidence on safe PT practice but also puts forward a protocol and pathway for HSCT rehabilitation, highlights the importance of individualized exercise intervention for HSCT patients, and outlines safe practice guidelines for the physical therapists working in this field.
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10.
Worldwide Network for Blood and Marrow Transplantation (WBMT) recommendations for establishing a hematopoietic stem cell transplantation program in countries with limited resources (Part II): clinical, technical and socio-economic considerations
Aljurf, M., Weisdorf, D., Hashmi, S. K., Nassar, A., Gluckman, E., Mohty, M., Rizzo, D., Pasquini, M., Hamadani, M., Saber, W., et al
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2019
Abstract
The development of hematopoietic stem cell transplantation (HSCT) programs can face significant challenges in most developing countries because such endeavors must compete with other government health care priorities, including the delivery of basic services. While this is may be a limiting factor, these countries should prioritize development of the needed expertise to offer state of the art treatments including transplantation, by providing financial, technological, legal, ethical and other needed support. This would prove beneficial in providing successful programs customized to the needs of their population, and potentially provide long-term cost-savings by circumventing the need for their citizens to seek care abroad. Costs of establishing HSCT program and the costs of the HSCT procedure itself can be substantial barriers in developing countries. Additionally, socioeconomic factors intrinsic to specific countries can influence access to HSCT, patient eligibility for HSCT and timely utilization of HSCT center capabilities. This report describes recommendations from the Worldwide Network for Blood and Marrow Transplantation (WBMT) for establishing HSCT programs with a specific focus on developing countries, and identifies challenges and opportunities for providing this specialized procedure in the resource constrained setting.