Blood cell therapy最新文献

{"title":"Successful hematopoietic stem cell transplantation with reduced dose of busulfan for Omenn syndrome.","authors":"Yukihiro Matsukawa,&nbsp;Kyohei Isshiki,&nbsp;Tomoo Osumi,&nbsp;Satoshi Fujiyama,&nbsp;Hiroko Fukushima,&nbsp;Toru Uchiyama,&nbsp;Masaki Yamada,&nbsp;Takao Deguchi,&nbsp;Ken-Ichi Imadome,&nbsp;Kimikazu Matsumoto,&nbsp;Daisuke Tomizawa,&nbsp;Hidetoshi Takada,&nbsp;Masafumi Onodera,&nbsp;Motohiro Kato","doi":"10.31547/bct-2021-021","DOIUrl":"https://doi.org/10.31547/bct-2021-021","url":null,"abstract":"<p><p>Omenn syndrome (OS) is typically observed in the autosomal recessive form of severe combined immunodeficiency (SCID) with autoreactive manifestations, and it requires allogeneic hematopoietic stem cell transplantation. Unlike non-OS SCID, a conditioning regimen is usually required to eradicate T-cells; however, optimal conditioning regimens are not established mainly because of the rarity of OS. Here, we report a case of hematopoietic stem cell transplantation with a reduced dose of busulfan, as a conditioning regimen and successful engraftment with complete chimerism. OS was diagnosed in a one-month-old boy based on a diffuse erythematous rash, absent B-cells, and activated T-cells. Genetic analysis failed to identify causative mutations for OS/SCID, such as <i>RAG1/2</i>. Bone marrow transplantation was performed from his HLA-matched sister with a conditioning regimen consisting of targeted busulfan, fludarabine, and anti-thymocyte globulin. Cyclosporine had been administered before transplantation to control abnormal T-cell activation and continued for graft-versus-host disease (GVHD) prophylaxis. Engraftment was achieved on day 12, and no GVHD symptoms were observed. For stem cell transplantation for OS, prior control of autoreactive symptoms with immunosuppressants is important for safe transplantation and reduced intensity conditioning (RIC) can be an option to achieve sustained engraftment.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 3","pages":"75-78"},"PeriodicalIF":0.0,"publicationDate":"2022-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a0/a2/2432-7026-5-3-0075.PMC9873423.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10584932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Successful treatment of acute pancreatitis associated with late-onset acute liver GVHD after second allogeneic hematopoietic cell transplantation.","authors":"Yuka Hosokawa,&nbsp;Tomomi Toubai,&nbsp;Rintaro Ohe,&nbsp;Masashi Hosokawa,&nbsp;Ryo Sato,&nbsp;Akane Yamada,&nbsp;Keiko Aizawa,&nbsp;Masahito Himuro,&nbsp;Satoshi Ito,&nbsp;Masakazu Yamamoto,&nbsp;Daniel Peltier,&nbsp;Kenichi Ishizawa","doi":"10.31547/bct-2021-023","DOIUrl":"https://doi.org/10.31547/bct-2021-023","url":null,"abstract":"<p><p>We report the case of a 28-year-old woman who developed upper abdominal pain and jaundice after a second unrelated allogeneic hematopoietic cell transplantation (allo-HCT) for acute lymphoid leukemia (ALL). Laboratory data showed elevated levels of liver enzymes, amylase, and lipase. Although acute pancreatitis was suspected, no structural lesions were detected. Liver biopsy was compatible with late-onset acute graft-versus-host disease (GVHD), which resolved following treatment with methylprednisolone (mPSL) and tacrolimus (TAC). In addition, her serum amylase level and abdominal pain rapidly resolved following acute GVHD-directed therapy. Acute pancreatitis concomitant with late-onset acute liver GVHD is extremely rare and has not been documented subsequent to a second allo-HCT.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 3","pages":"79-82"},"PeriodicalIF":0.0,"publicationDate":"2022-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4a/80/2432-7026-5-3-0079.PMC9873419.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10584934","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Trends in disease indications for hematopoietic stem cell transplantation in the Asia-Pacific region: A report of the Activity Survey 2017 from APBMT.","authors":"Minako Iida, Kaiyan Liu, Xiao Jun Huang, Wu Depei, Yachiyo Kuwatsuka, Joon Ho Moon, Anthony Dodds, Leonie Wilcox, Bor-Sheng Ko, Amir Ali Hamidieh, Kim Wah Ho, Artit Ungkanont, Aloysius Ho, Tasneem Farzana, Joycelyn Sim, Huynh Van Man, Mafruha Akter, Prasad Abeysinghe, Marjorie Rose Bravo, Aye Aye Gyi, Bishesh Sharma Poudyal, Khishigjargal Batshkh, Alok Srivastava, Shinichiro Okamoto, Yoshiko Atsuta","doi":"10.31547/bct-2022-002","DOIUrl":"10.31547/bct-2022-002","url":null,"abstract":"<p><p>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.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 4","pages":"87-98"},"PeriodicalIF":0.0,"publicationDate":"2022-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/06/77/2432-7026-5-4-0087.PMC9873430.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10585502","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adherence to long-term follow-up preventive practices in allogeneic hematopoietic cell transplantation survivors from North India.","authors":"Niranjan S Khaire, Prashant Chhabra, Dikshat G Gupta, Aditya Jandial, Alka Khadwal, Kripa Shanker Kasudhan, Shaweta Kaundal, Madhu Chopra, Arihant Jain, Gaurav Prakash, Navneet S Majhail, Pankaj Malhotra, Deepesh P Lad","doi":"10.31547/bct-2021-025","DOIUrl":"10.31547/bct-2021-025","url":null,"abstract":"<p><strong>Introduction: </strong>There are existing international guidelines for long-term follow-up (LTFU) care of allogeneic hematopoietic cell transplantation (allo-HCT) survivors. However, implementing these guidelines represents a unique challenge in resource-challenged settings.</p><p><strong>Methods: </strong>This study aimed to evaluate adherence to recommended surveillance in allo-HCT survivors at an academic center in North India and study the incidence of late effects. This single-center, retrospective study analyzed records of allo-HCT recipients from 2016 to 2020. Survivors were screened in our LTFU clinic at day +100 and +365 using cardiometabolic parameters (screening for hypertension, dyslipidemia, hyperglycemia, 24-hour urine protein, thyroid function), pulmonary function test (PFT), bone mineral density (BMD), and initiation of revaccination.</p><p><strong>Results: </strong>A total of 40/80 (50%) allo-HCT survivors were alive at a median of 888 days (IQR 515-1,306). The adherence to home-based screening parameters such as blood pressure and blood glucose was highest (>75%), followed by lab-based parameters (45-70%), and lowest for specialized tests such as PFT (<50%) at both day +100 and +365 time points. Adherence to the initiation of revaccination was only 67%. At least one cardiometabolic parameter was out of range in 55% and 63% of survivors at day +100 and +365, respectively.</p><p><strong>Conclusion: </strong>The adherence to recommended surveillance measures for allo-HCT survivors in an academic LTFU clinic at one year was only 75% overall. Cardiometabolic abnormalities were noted in more than half of the survivors. This study emphasizes the need for a structured LTFU clinic in all centers performing HCT.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 3","pages":"83-86"},"PeriodicalIF":0.0,"publicationDate":"2022-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/db/7b/2432-7026-5-3-0083.PMC9873420.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10584931","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Quality of life and informational needs for allogeneic hematopoietic stem cell transplant among patients and their caregivers visiting long-term follow-up clinic.","authors":"Shohei Nakajima,&nbsp;Kiyoko Kamibeppu","doi":"10.31547/bct-2021-005","DOIUrl":"https://doi.org/10.31547/bct-2021-005","url":null,"abstract":"<p><strong>Purpose: </strong>To examine the relationship between health-related quality of life (HRQOL) and fulfillment of informational needs among patients for allogeneic hematopoietic stem cell transplant (HSCT) and caregivers who visit long-term follow-up (LTFU) clinics within 1.5 years of post-HSCT.</p><p><strong>Methods: </strong>We conducted a cross-sectional survey at two university hospitals in Japan between May and December 2018 using self-administered questionnaires and medical records. Based on previous research and patient interviews, informational needs of patients and caregivers were categorized into general information, post-discharge treatments, side effects and complications, self-care, psychosocial problems, and social resources. The HRQOL of patients and caregivers was measured using the Japanese Functional Assessment of Cancer Therapy-Bone Marrow Transplant (for patients) and Caregiver Quality of Life Index-Cancer (for caregivers). In addition, the pooled-regression actor-partner interdependence model approach was employed to analyze the relationships using R ver.3.6.0.</p><p><strong>Results: </strong>A total of 16 patients and 14 caregivers were analyzed. The mean total score of the FACT-BMT was 91.0, and the mean total score of the Caregiver Quality of Life Index-Cancer was 88.2. For both patients and caregivers, fulfillment of informational needs regarding side effects and complications (estimates = 0.55, t (16) = 4.88, <i>P</i> < 0.001) and self-care (estimates = 0.73, t (13) = 5.02, <i>P</i> < 0.001) exerted actor effects on their HRQOL, whereas fulfillment of informational needs regarding psychosocial problems (estimates = 0.35, t (13) = 2.90, <i>P</i> = 0.012) exerted a partner effect on the mutual HRQOL.</p><p><strong>Conclusions: </strong>Multidimensional physio-psychosocial approaches toward patients and their caregivers are important to enhance their HRQOL during the acute phase after HSCT. Detailed overviews of and methods to cope with patients' psychosocial issues should be provided before discharge, especially for caregivers unable to visit the LTFU clinics.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 2","pages":"35-44"},"PeriodicalIF":0.0,"publicationDate":"2022-05-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/c3/cc/2432-7026-5-2-0035.PMC9870686.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10575312","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Nutritional support in allogeneic hematopoietic stem cell transplantation Asian perspective.","authors":"Shigeo Fuji,&nbsp;Jessica Cheng,&nbsp;Kimikazu Yakushijin,&nbsp;Chinadol Wanitpongpun","doi":"10.31547/bct-2021-024","DOIUrl":"https://doi.org/10.31547/bct-2021-024","url":null,"abstract":"<p><p>Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an integral part of the treatment strategy for patients with malignant or non-malignant hematological diseases. Clinical outcomes of patients undergoing allo-HSCT have significantly improved in recent decades. However, transplant-related morbidity and mortality remain major issues for allo-HSCT recipients. With regard to nutrition, patients undergoing allo-HSCT are at high risk for malnutrition. It is expected that clinical practice concerning nutritional support in allo-HSCT has been improving in recent decades; however, no data directly support this expectation. One major issue in managing nutritional support during allo-HSCT is the lack of large-scale randomized prospective studies, which leads to a lack of well-established strategies. Accordingly, we need to gather data from studies in non-HSCT and allo-HSCT settings. In some Asia-Pacific countries, a physician's lack of knowledge of nutritional support may impede the application of nutritional support practices recommended by existing guidelines. Another barrier may be the lack of access to an adequately qualified or trained registered dietitian (RD) at allo-HSCT units. Adequate training in the nutritional management of allo-HSCT patients should be provided to all RDs working with HSCT. Herein, we summarize the information on nutritional support in allo-HSCT, focusing on an Asian perspective.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 2","pages":"54-60"},"PeriodicalIF":0.0,"publicationDate":"2022-05-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/12/50/2432-7026-5-2-0054.PMC9870687.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10575309","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Allogeneic Hematopoietic Stem Cell Transplantation in Severe Aplastic Anemia: A Single Centre Experience in Malaysia.","authors":"Gilbert Wilfred,&nbsp;Tee Chuan Ong,&nbsp;Syed Abd Kadir Sh Shahnaz,&nbsp;Ho Kim Wah,&nbsp;Edmund Syed Carlo,&nbsp;Sathar Jameela,&nbsp;Sen Mui Tan","doi":"10.31547/bct-2021-018","DOIUrl":"https://doi.org/10.31547/bct-2021-018","url":null,"abstract":"<p><strong>Background: </strong>Hematopoietic stem cell transplantation (HSCT) provides curative therapy in almost 90% of patients with severe aplastic anemia (SAA). Older age, long duration of disease with consequent heavy exposure to transfusion, and active infection at the time of HSCT have a negative influence on the outcomes, causing graft failure (GF) and graft versus host disease (GVHD).</p><p><strong>Purpose: </strong>To describe the outcomes of all patients with SAA who received hematopoietic stem cell transplantation at a tertiary center in Malaysia.</p><p><strong>Materials and methods: </strong>We included a 20 y cohort of patients who underwent transplantation from January 1, 1999 to December 31, 2019. Data were obtained from electronic medical records. Demographics, clinical characteristics, and treatment outcomes were analyzed using descriptive statistics. Overall survival (OS) was analyzed using Kaplan-Meier curves. All analyses were conducted using the Statistical Package for the Social Sciences (SPSS) version 25.</p><p><strong>Results: </strong>Eighty patients were analyzed. The median age at diagnosis was 19 years, and 59% patients were male (n = 47). Malay ethnicity was the highest (52.5%), followed by Chinese (20.0%) and Native Sabah (15.0%). The median duration from diagnosis to transplantation was 13.5 weeks. A majority of patients received Cy-ATG conditioning (n = 51, 63.8%). Forty-one patients (51.2%) used peripheral blood as stem cell source, 36 patients (45.0%) used granulocyte colony stimulating factor (G-CSF) primed marrow graft and 3 patients (3.8%) used both. The mean nucleated mononuclear cell and CD34 cell doses were 4.7 ± 1.7 × 10⁸/kg and 4.6 ± 1.9 × 10⁶/kg, respectively. Median engraftment for WBCs and platelets was 14 and 15 days, respectively. There was no difference in WBC and platelet engraftment in patients who received peripheral blood stem cell transplantation or bone marrow transplant. At a median follow-up of 54 months, 49 patients (61.3%) achieved complete remission and 8 patients (10.0%) achieved partial remission. The estimated 5 y OS was 63% and higher among those who received HSCT within 3 months of diagnosis. Twenty-two patients (27.5%) died within 100 d of transplantation, and a majority of these died due to pre-engraftment death.</p><p><strong>Discussion and conclusions: </strong>Our study found that patients who received early allogeneic transplantation for SAA had better outcomes. Pre-engraftment failure was the major cause of transplant-related mortality within 100 d. Further studies are required to identify the factors responsible for delaying transplantation to improve treatment outcomes.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 2","pages":"45-53"},"PeriodicalIF":0.0,"publicationDate":"2022-05-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a4/c6/2432-7026-5-2-0045.PMC9870683.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10575311","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluating the Efficacy, Toxicity and Pharmacokinetic Profile of Oral Busulfan in Allogeneic Stem Cell Transplant Patients.","authors":"Ross Salvaris,&nbsp;Sam Salman,&nbsp;Sean O'Halloran,&nbsp;David Joyce,&nbsp;Navin Mathew,&nbsp;Julian Cooney,&nbsp;Matthew Wright,&nbsp;Paul Cannell,&nbsp;Duncan Purtill","doi":"10.31547/bct-2021-019","DOIUrl":"https://doi.org/10.31547/bct-2021-019","url":null,"abstract":"<p><strong>Background: </strong>Oral busulfan and intravenous cyclophosphamide (Bu/Cy) are common myeloablative preparations used in allogeneic hematopoietic stem cell transplantation (HSCT). Herein, we investigated the safety of (Bu/Cy) administration during HSCT.</p><p><strong>Methods: </strong>Patients administered Bu/Cy for allogeneic HSCT at Royal Perth Hospital and Fiona Stanley Hospital between 2007 and 2017 were reviewed for inclusion in the study. We performed busulfan pharmacokinetic (PK) testing for a subset of patients and allometric scaling modeling to assess the best method of busulfan dosing in patients at extremes of weight.</p><p><strong>Results: </strong>Sixty-nine patients were included in the clinical outcome analysis. The median follow-up period was 32 months (range, 9-114 months). The three-year overall survival rate was 62% (95% confidence interval (CI), 51%-75%), and transplant-related mortality was 4% at 6 months (95% CI, 1-7%), with a low rate of sinusoidal obstruction syndrome of the liver being observed. In addition, relapse was 38% (95% CI, 30%-44%) at 3 years. The PK information of 15 patients receiving busulfan was available after oral dosing. The average per-dose busulfan exposure was 1,350 μmol.min/L (range, 878-1,717 μmol.min/L), and the within target range was 1,000-1,500 μmol.min/L in 73% of patients. Of the size measures investigated, ideal and adjusted body weight (ABW40) provided the best fit. No association was observed between busulfan exposure, toxicity, and relapse.</p><p><strong>Conclusions: </strong>Overall, Bu/Cy administration appeared safe when dosed in relation to weight, showing a low early transplant-related mortality rate following adequate busulfan exposure in majority of the cases. Body size measures, such as ideal body weight or ABW40, are likely more suitable for use during busulfan dosing, particularly at high extremes of the body mass index classification.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 2","pages":"61-68"},"PeriodicalIF":0.0,"publicationDate":"2022-05-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/1d/86/2432-7026-5-2-0061.PMC9870685.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10575315","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Severe bloody diarrhea due to cytokine release syndrome after chimeric antigen receptor T cell therapy for refractory acute lymphoblastic leukemia.","authors":"Haruko Shima,&nbsp;Takahiro Ishikawa,&nbsp;Jumpei Ito,&nbsp;Katsura Emoto,&nbsp;Takumi Kurosawa,&nbsp;Dai Keino,&nbsp;Fumito Yamazaki,&nbsp;Hiroaki Goto,&nbsp;Hiroyuki Shimada","doi":"10.31547/bct-2021-009","DOIUrl":"https://doi.org/10.31547/bct-2021-009","url":null,"abstract":"<p><p>Cytokine release syndrome (CRS), which may be associated with fever, hypotension, hypoxia, and organ damage, is caused by a massive cytokine release after chimeric antigen receptor (CAR)-T cell therapy. We present the case of a patient who developed severe bloody diarrhea due to CRS after CAR-T cell infusion. A 10-year-old boy presented with a second relapse of B-cell precursor acute lymphoblastic leukemia 6 months after hematopoietic stem cell transplantation from an unrelated donor. CAR-T cells (tisagenlecleucel) were infused at the third complete remission after salvage chemotherapy. While fever >39°C was sustained from day 4, circulatory and respiratory status remained stable. However, he experienced severe bloody diarrhea. There was no evidence of infection; lower gastrointestinal (GI) endoscopy revealed extensive edema with erosion and ulceration, suggestive of non-specific intestinal inflammation. Thus, we considered CRS-associated grade 3 GI damage and administered a single dose of tocilizumab for grade 2 CRS, followed by 4 days of corticosteroids. Afterwards, no fever or GI bleeding was observed. Biopsy of the intestinal mucosa revealed ulcerative change with a lack of epithelial cells, which may correspond to histologic grade 4 graft versus host disease (GVHD). However, diarrhea corresponded to stage 1 GVHD, and the GVHD risk after CAR-T cell infusion has been reported to be rare in clinical practice. Although severe GI symptoms associated with CRS after CAR-T therapy are rare, early tocilizumab use is recommended for non-infectious severe GI symptoms to avoid long-term corticosteroid use, which may reduce CAR-T cell efficacy.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 1","pages":"31-34"},"PeriodicalIF":0.0,"publicationDate":"2022-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/ae/f5/2432-7026-5-1-0031.PMC9847265.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10585980","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pattern of autologous stem cell transplants at a tertiary care government hospital, with emphasis on transplant outcomes with pre-harvest CD34+ level.","authors":"Tuphan Kanti Dolai,&nbsp;Rajib De,&nbsp;Ankita Sen,&nbsp;Shuvra Neel Baul,&nbsp;Sumit Mitra,&nbsp;Subham Bhattacharya,&nbsp;Indrani Mondal,&nbsp;Kaushik Mukhopadhyay,&nbsp;Arnab Chattopadhyay,&nbsp;Shyamali Dutta,&nbsp;Prakas Kumar Mandal","doi":"10.31547/bct-2021-010","DOIUrl":"https://doi.org/10.31547/bct-2021-010","url":null,"abstract":"<p><strong>Purpose: </strong>Autologous stem cell transplantation (ASCT) is an established therapy for many hematological diseases. This study assessed the pattern of ASCTs at a tertiary care center and associated factors, including pre-harvest CD34+ stem cell levels, leading to improved engraftment outcomes.</p><p><strong>Methodology: </strong>A retrospective study was conducted in India, between February 2009-August 2020. Patients who underwent ASCT for different hematological malignancies (n=65) were included, and the patients' age, sex, type and stage of disease, pre- and post-harvest CD34+ counts, and time to attain platelet/neutrophil engraftment or febrile neutropenia were analyzed. The post-harvest CD34+ dose was calculated. Pre-conditioning was performed using Granulocyte Colony Stimulating Factor (GCSF)±Plerixafor. Progression-free survival (PFS) was calculated using relapse/death as the endpoint.</p><p><strong>Results: </strong>The median age of the cohort (n=65) was 49 years, with a male preponderance. Multiple myeloma was the most common malignancy (70.8% [46/65]), requiring ASCT. The median time to ASCT was 13 months. All patients had received GCSF, while Plerixafor was used in 17 patients with a pre-harvest CD34+ count of <10 cells/μL. The median pre-harvest CD34+ concentration and post-harvest CD34+ cell dose was 27.54 cells/μL (n=26) and 5.23×10⁶ cells/kg body weight (n=65), respectively. The median time to engraftment was 11 and 12 days, for neutrophils and platelets, respectively. One patient did not engraft and was excluded from the analysis. The time required to attain neutrophil engraftment was significantly lower (<i>p</i>=0.02) among freshly harvested stem cells (n=48) than that of cryopreserved products (n=17). Platelet engraftment associated with CD34+ pre- and post-harvest levels was not significant (<i>p</i>=0.06). The time to attain neutropenia and subsequent febrile neutropenia was significantly lower with an adequate post-harvest CD34+ dose (<i>p</i>=0.009). Febrile neutropenia was seen in 83.1% (54/65) patients. The median time for febrile neutropenia was 4 days post-ASCT. Pre- and post-harvest CD34+ concentrations were directly proportional to each other (<i>p</i><0.001). The median PFS was 112 months (n=65). Survival was better in males (median PFS: 112 months) vs. females (median PFS: 59 months) (<i>p</i>=0.27). Eight patients relapsed, and eight patients had died.</p><p><strong>Conclusion: </strong>Although unrelated to age or sex, the post-harvest CD34+ dose was inversely related to febrile neutropenia. As pre- and post-harvest CD34+ levels were directly proportional, pre-harvest CD34+ concentrations may be reliably used to assess engraftment outcomes. Rapid neutrophil engraftment was noted in fresh stem cells with PFS of 112 months, and was better among males, the exact reason being unknown. Thus, a larger number of patients should be followed up to obtain an accurate picture.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 1","pages":"16-26"},"PeriodicalIF":0.0,"publicationDate":"2022-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/76/e2/2432-7026-5-1-0016.PMC9847276.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10640714","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}