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[Stem cell transplantation unit: Guidelines from the francophone Society of bone marrow transplantation and cellular therapy (SFGM-TC)].
Faucher, C, Adam, C, Bancillon, N, Bertrand, E, Colledani, F, de Berranger, E, Denis, V, Girard, I, Hamzy, F, Loukili, N, et al
Bulletin du cancer. 2019;(1S):S1-S9
Abstract
Allogeneic hematopoietic cell transplantation (HCT) is part of the standard of care for many hematological diseases. Over the last decades, significant advances in patient and donor selection, conditioning regimens as well as supportive care of patients undergoing allogeneic HCT leading to improved overall survival have been made. In view of many new treatment options in cellular and molecular targeted therapies, the place of allogeneic transplantation in therapy concepts must be reviewed. Most aspects of HCT are well standardized by national guidelines or laws as well as by certification labels such as FACT-JACIE. However, the requirements for human resources, construction and layout of a unit treating patients during the transplantation procedure and for different complications are not well defined. Here, we describe the process of planning a transplant unit in order to open a discussion that could lead to more precise guidelines in the field of personnel and infrastructural requirements for hospitals caring for people with severe immunosuppression.
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High serum neurofilament light chain normalizes after hematopoietic stem cell transplantation for MS.
Thebault, S, R Tessier, D, Lee, H, Bowman, M, Bar-Or, A, Arnold, DL, L Atkins, H, Tabard-Cossa, V, Freedman, MS
Neurology(R) neuroimmunology & neuroinflammation. 2019;(5):e598
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Abstract
OBJECTIVE To evaluate neurofilament light chain (NfL) levels in serum and CSF of patients with aggressive MS pre- and post-treatment with immunoablation followed by autologous hematopoietic stem cell transplantation (IAHSCT) and examine associations with clinical and MRI outcomes. METHODS Paired serum and CSF in addition to MRI and clinical measures were collected on 23 patients with MS at baseline and 1 and 3 years post-IAHSCT. An additional 33 sera and CSF pairs were taken from noninflammatory neurologic controls. NfL levels were quantitated using the Simoa platform (Quanterix). RESULTS Baseline MS NfL levels were significantly elevated relative to controls in serum (p = 0.001) and CSF (p = 0.001). Following IAHSCT, high pretreatment NfL levels significantly reduced in serum (p = 0.0023) and CSF (p = 0.0068) and were not significantly different from controls. Serum and CSF NfL levels highly correlated (r = 0.81, p < 0.0001). Baseline NfL levels were associated with worse pretreatment disease measures (Expanded Disability Status Scale [EDSS], relapses, MRI lesions, and MR spectroscopy (MRS) N-acetylaspartate/creatine). Elevated baseline NfL levels were associated with persistently worse indices of disease burden post-IAHSCT (sustained EDSS progression, cognition, quality of life, T1 and T2 lesion volumes, MRS, and brain atrophy). CONCLUSION These data substantiate that serum and CSF NfL levels reflect disease severity and treatment response in patients with MS and may therefore be a useful biomarker. Baseline serum levels associated with markers of pretreatment disease severity and post-treatment outcomes. CLASSIFICATION OF EVIDENCE This study provides Class II evidence that for patients with aggressive MS, serum NfL levels are associated with disease severity.
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Meeting the needs of CML patients in resource-poor countries.
Malhotra, H, Radich, J, Garcia-Gonzalez, P
Hematology. American Society of Hematology. Education Program. 2019;(1):433-442
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Abstract
Subsequent to the development and global availability of BCR/ABL-targeted tyrosine kinase inhibitors (TKIs), the prognosis of patients with chronic myeloid leukemia (CML), at least those in the chronic phase, has markedly improved, and in the developed world, the average lifespan of these patients is now close to that of age- and sex-matched subjects without the disease. However, the situation in low- and middle-income countries (LMICs) may not be so rosy. Many important differences in hematological cancers, including CML, have been highlighted in various publications in LMICs vs developed countries. These include differences in incidence and prevalence rates, age and stage of disease at diagnosis, response rates, and survival. Some of the possible reasons proposed for these are varying socioeconomic milieu (impacting availability of effective drugs and essential monitoring), environmental factors (mainly exposure to viral infections and pesticides), nutritional factors with interplay of malnutrition and diet on drug absorption and blood levels, and possible unknown genetic factors. Although generic first-generation TKIs (imatinib) are available in many parts of the world, several challenges remain in providing optimal treatment to patients with CML in resource-poor countries. Some of these include availability of optimal and high-quality BCR/ABL testing, availability and expense related to use of second- and third-generation TKIs (nilotinib, dasatinib, bosutinib, and ponatinib) and hematopoietic stem cell transplantation, issues with compliance and toxicities of drugs, and ensuring a minimal standard-of-care treatment and monitoring for every patient diagnosed with CML. For the purpose of this article, the more objective country label-LMIC-coined by the World Bank will be used (gross national income per capita between $1026 and $3995; World Bank, June 2019). Some of these issues will be discussed in this article in greater detail by experts in the field in 3 different but interconnected sections.
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Non-Graft-versus-Host Disease Ocular Complications after Hematopoietic Cell Transplantation: Expert Review from the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research and the Transplant Complications Working Party of the European Society for Blood and Marrow Transplantation.
Inamoto, Y, Petriček, I, Burns, L, Chhabra, S, DeFilipp, Z, Hematti, P, Rovó, A, Schears, R, Shah, A, Agrawal, V, et al
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2019;(5):e145-e154
Abstract
Non-graft-versus-host disease (GVHD) ocular complications are generally uncommon after hematopoietic cell transplantation (HCT) but can cause prolonged morbidity affecting activities of daily living and quality of life. Here we provide an expert review of non-GVHD ocular complications in a collaboration between transplantation physicians and ophthalmologists through the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research and the Transplant Complications Working Party of the European Society of Blood and Marrow Transplantation. Complications discussed in this review include cataracts, glaucoma, ocular infections, ocular involvement with malignancy, ischemic microvascular retinopathy, central retinal vein occlusion, retinal hemorrhage, retinal detachment and ocular toxicities associated with medications. We summarize the incidence, risk factors, screening, prevention, and treatment of individual complications and generate evidence-based recommendations. Baseline ocular evaluation before HCT should be considered in all patients who undergo HCT. Follow-up evaluations should be considered according to clinical signs and symptoms and risk factors. Better preventive strategies and treatments remain to be investigated for individual ocular complications after HCT. Both transplantation physicians and ophthalmologists should be knowledgeable about non-GVHD ocular complications and provide comprehensive collaborative team care.
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Allogeneic transplantation in high-risk chronic lymphocytic leukemia: a single-center, intent-to-treat analysis.
Hoffmann, A, Dietrich, S, Hain, S, Rieger, M, Hegenbart, U, Sellner, L, Ho, AD, Müller-Tidow, C, Dreger, P
Haematologica. 2019;(7):e304-e306
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Effects of exercise in combination with autologous bone marrow stem cell transplantation for patients with type 1 diabetes.
Mohamed, MT, Embaby, EA, Labib, A, El-Husseiny, M, Khamis, H, El-Demery, A, Shoukry, MM
Physiotherapy theory and practice. 2019;(12):1233-1242
Abstract
Stem cell therapy is a promising approach for the treatment of type 1 diabetes mellitus (T1D). Previous studies recommended regular exercise for the control of T1D. Experimental studies showed that a combination of stem cells and exercise yielded a better outcome. Yet, the effect of exercise programs following stem cell transplantation in patients with T1D has not been investigated. Thus, the current study aimed to examine the effect of a combined exercise program on measures of glycemic control in patients with T1D who received autologous bone marrow stem cell transplantation (ABMSCT). Thirty patients with controlled T1D were assigned into two equal groups. Both groups underwent ABMSCT and received insulin therapy and a diabetic diet regime. Only the exercise group followed the combined exercise program. Outcome measures of glycemic control (i.e. fasting blood glucose level [FBG], post-prandial blood glucose level [PPG], HbA1c, daily insulin dosage, and C-peptide levels) were tested before and after a 3-month rehabilitation period. There were significant (p < 0.05) decreases in all outcome measures except C-peptides after ABMSCT compared with before in both groups. Moreover, there was a significant decrease in the mean value of HbA1c in the exercise group compared with the control group after rehabilitation. Overall, this study strengthens the idea that adding exercise to ABMSCT is important to help control diabetes in patients with T1D.
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The effect of three different solutions on preventing oral mucositis in cancer patients undergoing stem cell transplantation: a non-randomized controlled trial: A Turkish study - NON-RANDOMISED TRIAL.
Harman, M, Ovayolu, N, Ovayolu, O
JPMA. The Journal of the Pakistan Medical Association. 2019;(6):811-816
Abstract
OBJECTIVE To evaluate the effect of different solutions administered to patients undergoing stem cell transplantation on oral mucositis. METHODS The non-randomised controlled trial was conducted at a Istanbul Medipol Mega university hospital in Turkey between May 2014 and June 2016, and comprised patients undergoing stem cell transplantation. They were divided into three groups. Group 1 had patients using chlorhexidine gluconate and benzydamine hydrochloride solution. Group 2 had those using calcium and phosphate solution. Group 3 patients were using black mulberry syrup. Data was collected using a structured questionnaire and the World Health Organisation mucositis assessment scale. Assessment was done on days 7, 14 and 21. Clinical significance of oral solutions was statistically determined. RESULTS Of the 83 patients, 30(36%) were in group 1, 28(34%) in group 2, and 25(30%) in group 3. On day 7, there was no significant difference in terms of grades among the groups (p>0.05). On day 14, grade 2 mucositis was seen in 2(8%) patents in group 3, 5(17.9%) in group 2 and 5(16.7%) in group 1; Grade 3 mucositis was seen in 2(6.7%) patients in group 1, but none in the other two groups. On day 21, grade 3 mucositis was present in 2(8.0%) in group 3, 2(7.1%) in group 2, and 4(13.3%) in group 1. CONCLUSIONS The use of black mulberry and calcium-phosphate solutions was found to be beneficial in preventing and treating oral mucositis.
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Treatment of MDS.
Platzbecker, U
Blood. 2019;(10):1096-1107
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Abstract
The heterogeneous nature of myelodysplastic syndromes (MDS) demands a complex and personalized variety of therapeutic approaches. Among them, allogeneic hematopoietic stem cell transplantation remains the only potentially curative option and is accessible to only a small number of fit patients. For the majority of patients with MDS, treatment strategies are nonintensive and risk-adapted (by the revised version of the International Prognostic Scoring System), ranging from iron chelation and growth factors to lenalidomide and hypomethylating agents. These approaches are noncurative and aimed instead at improving cytopenias and quality of life and delaying disease progression. These limitations underpin the need for more translational research-based clinical trials in well-defined subgroups of patients with MDS. Indeed, much progress has been made over the past decade in understanding the complex molecular mechanisms underlying MDS. Unfortunately, this has not yet translated into approval of novel treatment options. There is a particularly urgent medical need in patients failing current first-line therapies, such as with erythropoiesis-stimulating or hypomethylating agents. Nevertheless, actual developments are expected to pave the way for exciting novel therapeutic opportunities. This review provides an overview of the current therapeutic landscape in MDS focusing on recent advances in clinical and translational research.
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Vitamin D Modifies the Incidence of Graft-versus-Host Disease after Allogeneic Stem Cell Transplantation Depending on the Vitamin D Receptor (VDR) Polymorphisms.
Carrillo-Cruz, E, García-Lozano, JR, Márquez-Malaver, FJ, Sánchez-Guijo, FM, Montero Cuadrado, I, Ferra I Coll, C, Valcárcel, D, López-Godino, O, Cuesta, M, Parody, R, et al
Clinical cancer research : an official journal of the American Association for Cancer Research. 2019;(15):4616-4623
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Abstract
PURPOSE The biologically active metabolite of vitamin D3, 1,25-dihydroxyvitamin D3 (vit D), has immunoregulatory properties via binding vitamin D receptor (VDR). In a prospective trial, we previously reported a reduction in the incidence of chronic GvHD (cGvHD) among patients who received vit D after allogeneic stem cell transplantation (allo-HSCT; Clinical Trials.gov: NCT02600988). Here we analyze the role of patients and donors' VDR SNPs on the immunomodulatory effect of vit D. PATIENTS AND METHODS Patients undergoing allo-HSCT were included in a prospective phase I/II clinical trial (Alovita) in three consecutive cohorts: control (without vit D), low-dose (1,000 IU/day), and high-dose (5,000 IU/day) groups. Vit D was given from day -5 until +100 after transplant. Genotyping of four SNPs of the VDR gene, FokI, BsmI, ApaI, and TaqI, were performed using TaqMan SNP genotyping assays. RESULTS We observed a decrease in the incidence of overall cGvHD at 1 year after allo-HSCT depending on the use or not of vit D among patients with FokI CT genotype (22.5% vs 80%, P = 0.0004) and among those patients without BsmI/ApaI/TaqI ATC haplotype (22.2% vs 68.8%, P = 0.0005). In a multivariate analysis, FokI CT genotype significantly influenced the risk of cGvHD in patients treated with vit D as compared with the control group (HR 0.143, P interaction < 0.001). CONCLUSIONS Our results show that the immunomodulatory effect of vit D depends on the VDR SNPs, and patients carrying the FokI CT genotype display the highest benefit from receiving vit D after allo-HSCT.
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Pathology of Gastrointestinal and Liver Complications of Hematopoietic Stem Cell Transplantation.
Mourad, N, Michel, RP, Marcus, VA
Archives of pathology & laboratory medicine. 2019;(9):1131-1143
Abstract
CONTEXT.—: Despite advances in therapeutic and preventive measures, hematopoietic stem cell transplant recipients remain at risk for a variety of gastrointestinal and liver complications. OBJECTIVE.—: To detail the pathologic features of the various gastrointestinal and liver complications occurring after hematopoietic stem cell transplantation in relation to their clinical context. The specific complications covered include graft-versus-host disease, mycophenolate mofetil-induced injury, timeline of infections, neutropenic enterocolitis, gastrointestinal thrombotic microangiopathy, sinusoidal obstruction syndrome, hepatic iron overload, and the controversy around cord colitis syndrome. DATA SOURCES.—: The content of this article is based on pertinent peer-reviewed articles in PubMed, relevant textbooks, and on the authors' personal experiences. CONCLUSIONS.—: The final histopathologic diagnosis requires the integration of clinical and histologic findings and the exclusion of other competing causes of injury. Review of the clinical data, including the original disease pretransplant, the type of transplant, the timing of the gastrointestinal and/or liver manifestations, the timing of the biopsy after transplant, the presence of graft-versus-host disease in other organs and sites, the list of drug regimens, and the clinical and laboratory evidence of infection, is the key to reaching the proper histologic diagnosis.