Blood cell therapy最新文献

{"title":"Efficacy and safety of plerixafor in pediatric cancer patients undergoing peripheral blood stem cell harvest for autologous hematopoietic stem cell transplant","authors":"","doi":"10.31547/bct-2022-022","DOIUrl":"https://doi.org/10.31547/bct-2022-022","url":null,"abstract":"Plerixafor for peripheral blood hematopoietic stem cell (PB HSC) mobilization in children undergoing autologous hematopoietic stem cell transplantation is primarily used following failure of the initial mobilization attempt. Data on plerixafor use in pediatric patients are limited. This retrospective study conducted at a single tertiary care center in India, details the efficacy and safety of plerixafor for 10 children with relapsed/refractory solid tumors or lymphomas. High risk neuroblastomas (HR NB) underwent autologous HSCT as part of consolidation. Plerixafor was administered at a dose of 240 μg/kg body weight of the recipient, subcutaneously, approximately 11-12 h prior to harvest. Ten patients (eight males, two females), with a median age of 8 years (range 2-18 years), received plerixafor prior to PB HSC harvest. All patients were administered granulocyte colony stimulating factor (GCSF) before the administration of plerixafor. The median CD 34 count for all patients pre-plerixafor was 29/μL, nine patients exhibited higher CD 34 post plerixafor (median of 148/μL). In nine patients, the values of the CD 34 count and total leukocyte count (TLC) of the harvested product were available, and in all cases, we achieved a good yield. All patients in this study were heavily chemotherapy pre-treated, and the use of plerixafor resulted in a satisfactory yield of peripheral blood stem cells. No side effects were observed.","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"10 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135401537","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Factors Impacting Return to Work/School among Hematopoietic Cell Transplantation Survivors in India","authors":"","doi":"10.31547/bct-2023-017","DOIUrl":"https://doi.org/10.31547/bct-2023-017","url":null,"abstract":"Introduction : While there are data about return to work after hematopoietic cell transplantation (HCT) in survivors from resource-rich regions, similar data from resource-challenged settings are scarce. This study assessed the incidence of and factors affecting return to work/school (RTW) among HCT survivors in India. Methods : This single-center cross-sectional study was conducted at the long-term follow-up (LTFU) clinic of a large-volume HCT center during 2022-2023. HCT survivors surviving beyond four months were included after obtaining informed consent. Patients’ sociodemographic, disease, HCT, and work details were recorded. The factors affecting RTW were evaluated using univariate (ANOVA) and logistic regression analyses. Results : A total of 126 HCT survivors participated in the study. Of these, 34 (27%) did not RTW, 47 (37%) returned to part-time work, and 45 (36%) returned to full-time work at a median of more than three years post-HCT. The three groups did not significantly differ in age, sex, or marital status. The univariate analysis revealed that education, pre-HCT job status, income, and conditioning intensity were significantly associated with RTW. Logistic regression analysis revealed that survivors with a higher (taxable) income were more likely to RTW than those with a lower (non-taxable) income (OR 3.5; CI 1.2-10.2, p =0.01). Survivors with a desk job were more likely to RTW than those who were unemployed/retired or students (OR 4.5; CI 1.1-18.0, p =0.03). Conclusion : Socioeconomic factors, like pre-HCT job status and income, were significantly associated with post-HCT RTW. Therefore, there is a need to integrate multidisciplinary RTW programs for HCT survivors in India.","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"25 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135758448","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Three cases of suspected pleuroparenchymal fibroelastosis after allogeneic hematopoietic cell transplantation","authors":"","doi":"10.31547/bct-2023-018","DOIUrl":"https://doi.org/10.31547/bct-2023-018","url":null,"abstract":"This article reports the clinical course and imaging findings of three cases of suspected pleuroparenchymal fi-broelastosis (PPFE) after allogeneic hematopoietic cell transplantation (HCT). All patients complained of dyspnea more than 5 years after HCT, had progressive restrictive deficits on respiratory function tests, and presented with pneumothorax, pleural thickening, or exacerbation of consolidation in the upper lobe of the lung. Though lung biopsy was not done in all three cases, the clinical findings and results of spirometry were compatible with those of PPFE. PPFE has been sporadically reported as a pulmonary complication of allogeneic HCT; however, clinical diagnostic criteria other than histological diagnosis and treatment methods have not yet been established. The accumulation of more cases is necessary to improve the prognosis of PPFE complications.","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"44 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135758602","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Alternate Donor Transplantation.","authors":"William Yk Hwang,&nbsp;Piyanuch Kongtim,&nbsp;Navneet S Majhail,&nbsp;Ming Yao","doi":"10.31547/bct-2022-012","DOIUrl":"https://doi.org/10.31547/bct-2022-012","url":null,"abstract":"<p><p>There is a significant need for alternative donors other than full-matched related or unrelated donors for allogeneic hematopoietic stem cell transplantation, especially in the Asia Pacific, where donor registries are smaller, and ethnicities are far more diverse. Both umbilical cord blood (UCB) and haploidentical transplantation can be carried out despite significant human leukocyte antigen (HLA) mismatches between patients and donors and help to meet this need. There are advantages and disadvantages to UCB and haploidentical transplantation, though enhancements in technology continue to improve outcomes in both. Donor selection for these cell sources is dependent on the presence of donor specific anti-HLA antibodies in the recipient's serum, degree and characteristics of donor-recipient HLA mismatches, ABO compatibility. Specific to haploidentical transplantation, additional factors like donor age, sex, donor-recipient CMV serology as well as NK cell alloreactivity are also important.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 Spec Edition","pages":"S6-S14"},"PeriodicalIF":0.0,"publicationDate":"2022-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/74/ad/2432-7026-5-S-S0006.PMC10200364.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9569489","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":"The activities and regulatory landscape of cellular therapies including hematopoietic cell transplantation in the world.","authors":"Shinichiro Okamoto,&nbsp;Miguel-Angel Perales,&nbsp;Anna Sureda,&nbsp;Dietger Niederwieser","doi":"10.31547/bct-2022-013","DOIUrl":"https://doi.org/10.31547/bct-2022-013","url":null,"abstract":"<p><p>A variety of cellular therapies including hematopoietic cell transplantation (HCT) hold the promise to treat medical conditions and diseases that currently have limited or no effective therapeutic options. A number of cellular therapies other than HCT, such as CAR T-cell therapy, are currently in preclinical and clinical development and the field is rapidly growing. The current activity of cellular therapies, including HCT, in the clinical setting are summarized in this article. Collaborative efforts from all relevant professionals and organizations will be of great importance to overcome substantial challenges in clinical development and post-launch evidence collection of cellular therapies. Harmonization among decision-makers also plays a critical role in reinforcing consistency and improving efficiencies of the regulatory and health technology assessment process. For the long-term safety follow-up of patients undergoing cellular therapies, registries for HCT are able to manage the complexity of data and in the best position to introduce and monitor future innovative cellular therapies for a variety of hematological disorders.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 Spec Edition","pages":"S15-S24"},"PeriodicalIF":0.0,"publicationDate":"2022-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/77/11/2432-7026-5-S-S0015.PMC10200362.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9515730","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":"Clinical roles of TIM-3 in myeloid malignancies and its importance in cellular therapy.","authors":"Yoshikane Kikushige","doi":"10.31547/bct-2022-010","DOIUrl":"https://doi.org/10.31547/bct-2022-010","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML), one of the most common hematological malignancies worldwide, is derived from a fraction of stem cells known as leukemic stem cells (LSCs), which possess self-renewal and high propagation capacities. Remaining quiescent and being resistant to conventional chemotherapy, residual LSCs after chemotherapy drive leukemia regrowth, leading to AML relapse. Therefore, the eradication of LSCs is critical for the treatment of AML. We previously identified hepatitis A virus cellular receptor 2 (HAVCR2/TIM-3) as an LSC-specific surface molecule by comparing gene expression in LSCs and hematopoietic stem cells (HSCs). TIM-3 expression clearly discriminated LSCs from HSCs within the CD34⁺CD38^- stem cell fraction. Furthermore, AML cells secrete galectin-9, a TIM-3 ligand, in an autocrine manner, leading to constitutive TIM-3 signaling that maintains the self-renewal capacity of LSCs via the induction of β-catenin accumulation. Thus, TIM-3 is an indispensable functional molecule for human LSCs. Herein, we review the functional aspects of TIM-3 in AML and evaluate minimal/measurable residual disease with a focus on CD34⁺CD38^-TIM-3⁺ LSCs. Using sequential genomic analysis of identical patients, we determined that CD34⁺CD38^-TIM-3⁺ cells in the complete remission (CR) phase after allogeneic stem cell transplantation (allo-SCT) are the LSCs responsible for AML relapse. We retrospectively evaluated the incidence of TIM-3⁺ residual LSCs. All analyzed patients achieved CR and complete donor chimerism at the engraftment phase; however, the high frequency of residual TIM-3⁺ LSCs within the CD34⁺CD38^- fraction at engraftment was a significant and independent risk factor for relapse. Residual TIM-3⁺ LSC levels in the engraftment phase had a stronger impact on relapse than did pre-SCT disease status. Therefore, the evaluation of residual TIM-3⁺ LSCs is a promising approach for predicting leukemia relapse after allo-SCT.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 Spec Edition","pages":"S1-S5"},"PeriodicalIF":0.0,"publicationDate":"2022-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/82/fc/2432-7026-5-S-S0001.PMC10200361.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9569491","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":"Advances in Allogeneic Hematopoietic Stem Cell Transplantation for Acute Leukemia.","authors":"Yasuhito Nannya, Auro Viswabandya, Peihua Lu","doi":"10.31547/bct-2022-015","DOIUrl":"10.31547/bct-2022-015","url":null,"abstract":"<p><p>In acute leukemia, advances have been made in therapeutic strategies centered on allogeneic hematopoietic stem cell transplantation (allo-SCT), three of which are presented here. The indication of allo-SCT for acute myeloid leukemia (AML) in 1^st complete remission (CR1) has been debated. Genomic medicine has helped us gain a deeper understanding of this disease, some of which may serve as prognostic factors. Such genetic abnormalities could also help measure minimal residual disease (MRD) and provide additional clues to estimate the efficacy of chemotherapy. Combined with existing prognostic factors, these data can be used to construct a more accurate prognostic model, providing an optimal indication of allo-SCT for AML in CR1. Furthermore, overall treatment algorithms for high-risk AML after allo-SCT should include prophylactic and pre-emptive treatment to prevent relapse. These include immunotherapy using donor lymphocyte infusion (DLI), FLT3 inhibitors in <i>FLT3</i>-mutated AML, hypomethylating agents, or a combination of DLI with these agents. Clinical trials are currently ongoing to elucidate the role of these strategies, which will lead to a risk-adapted treatment for preventing relapse in high-risk AML. CD19-targeted chimeric antigen receptor (CAR) T-cell therapy induces a remarkable response in B-acute lymphoid leukemia (B-ALL); however, relapse remains a major problem. In this regard, allo-SCT as a consolidation treatment after CAR-T cell therapy for B-ALL is recommended for pediatric and adult patients. Achieving complete remission (CR) with CAR-T cell therapy is considered a promising bridging therapy to allo-SCT. Novel CAR-T treatment techniques are being developed to change their role as a pre-transplant treatment.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 Spec Edition","pages":"S25-S33"},"PeriodicalIF":0.0,"publicationDate":"2022-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/62/87/2432-7026-5-S-S0025.PMC10200363.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9521494","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":"Human leukocyte antigen allele and haplotype frequencies in Singapore bone marrow donors and cord blood units.","authors":"Alvin Ng Yu-Jin,&nbsp;Grace Benjamin Moshi,&nbsp;Arun Prasath,&nbsp;Marieta Chan,&nbsp;Poon Limei Michelle,&nbsp;Loh Chee Khiong Charles,&nbsp;Louise Cho,&nbsp;Valerie Voon,&nbsp;Seng Zi Jing,&nbsp;Phang Chew Yen,&nbsp;Aloysius Yew Leng Ho","doi":"10.31547/bct-2022-004","DOIUrl":"https://doi.org/10.31547/bct-2022-004","url":null,"abstract":"<p><p>We describe the allele and haplotype frequencies seen in a volunteer unrelated bone marrow donor registry, a public cord blood bank, and donor/recipient samples processed by the Health Sciences Authority (HSA) in Singapore. Historical human leukocyte antigen (HLA) typing reports were anonymized and combined. They were checked for HLA typing nomenclature discrepancies or ambiguities using the HLA-net UNIFORMATE tool, and for analysis, the validated data were subsequently separated into Chinese, Malay, Indian, and \"Others,\" according to the race classification system used in Singapore. Individual ethnic allele and haplotype frequencies were calculated with the HLA-net GENE[RATE] pipeline using basic statistics. The Basic Statistics Tool of HLA-net was used to estimate haplotype frequency using an expectation maximization algorithm, given a set of multi-allelic data pairs for a given HLA locus. The outputs downloaded from the site comprised plain text files with haplotype frequency estimates, results of a global linkage disequilibrium test, and standardized residuals (stdres) corresponding to deviations from expected frequencies. HLA typing results from 59,186 individuals met the inclusion criteria, yielding 118,372 analyzable alleles. In our study population, the haplotype A*33:03-B*58:01-C*03:02-DRB1*03:01～DQB1*02:01:01G with a frequency of 4.91% was the most common. This haplotype was also the most common among Singaporean Chinese donors. Consistent with the predominant Chinese population, haplotypes with a frequency greater than 1% were also the most frequently observed haplotypes in the Singaporean population. In the Malay donor population, the most common haplotype was A*33:03～B*44:03～C*07:01:01G～ DRB1*07:01-DQB1*02:01:01G, with a frequency of 3.41%, whereas within the Indian donor population, the most common haplotype was A*01:01-B*57:01-C*06:02～DRB1*07:01-DQB1*03:03, with a frequency of 3.42%. Haplotype diversity and composition statistics within donor pools provide HLA background data required for the targeted recruitment of donors to support the hematopoietic stem cell donor requirements of the country. These data may be used in the future to devise donor recruitment strategies for optimizing the donor pool through targeted publicity and accruals.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 4","pages":"99-106"},"PeriodicalIF":0.0,"publicationDate":"2022-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4d/87/2432-7026-5-4-0099.PMC9873421.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10590499","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":"Effect of Early Nutritional Support on Quality of Life by EORTC QLQ-C30 in Allogeneic Hematopoietic Stem Cell Transplantation.","authors":"Ayaka Inden,&nbsp;Takayoshi Tsukahara,&nbsp;Eiko Tachibana,&nbsp;Yasuyuki Nagata,&nbsp;Takaaki Ono,&nbsp;Akihiko Kato","doi":"10.31547/bct-2022-007","DOIUrl":"https://doi.org/10.31547/bct-2022-007","url":null,"abstract":"<p><strong>Purpose: </strong>Increasing attention is being paid to the importance of nutritional management of allogeneic hematopoietic stem cell transplant (allo-HSCT) patients. However, few studies have conducted detailed evaluations of both nutritional intake and quality of life (QOL) in allo-HSCT patients. Therefore, we investigated the nutritional status and quality of life of our allo-HSCT patients.</p><p><strong>Methods: </strong>The subjects were 26 adults who underwent allo-HSCT at Hamamatsu University Hospital between August 2018 and October 2021. Early nutritional intervention was provided from the time of the decision to perform allo-HSCT to the time of discharge, and it incorporated regular QOL assessments. The analyzed indices were nutritional intake, anthropometric measurements, body mass index (BMI), grip strength, body composition analyzer (InBody S10) measurements, and blood laboratory values including transthyretin levels. QOL was assessed using the QLQ-C30 questionnaire of the European Organization for Research and Treatment of Cancer (EORTC) (version 3.0) and calculated according to the EORTC scoring manual. The indices were compared at pre-transplantation, 30 days post-transplantation, 60 days post-transplantation, and at discharge. The association between pre-transplantation nutritional status and QOL was examined.</p><p><strong>Results: </strong>The median hospital stay after transplantation was 97 days (range, 78-123 days). Energy intake was maintained at 31 kcal/day/kg through 30 days post-transplantation, 60 days post-transplantation, and discharge, and protein intake was maintained at 1.0 g/day/kg throughout all time periods. There was a significant positive correlation between the pre-transplantation transthyretin level and the 60-day post-transplantation QOL scores for \"global health\", \"physical functioning\", \"cognitive functioning\", and \"emotional functioning\", and there were significant negative correlations with \"fatigue\" and \"pain\" that indicated improvement.</p><p><strong>Conclusion: </strong>Early nutritional management of allo-HSCT patients prior to transplantation allowed maintenance of nutritional intake, and higher pre-transplant transthyretin levels were associated with higher QOL scores at 60 days post-transplantation.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 4","pages":"107-115"},"PeriodicalIF":0.0,"publicationDate":"2022-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/ae/07/2432-7026-5-4-0107.PMC9873424.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10585496","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":"Hematopoietic Stem Cell Transplantation is a cost-effective alternative to enzyme replacement therapy in Gaucher Disease.","authors":"Fouzia N Aboobacker,&nbsp;Uday P Kulkarni,&nbsp;Anu Korula,&nbsp;Anup J Devasia,&nbsp;Sushil Selvarajan,&nbsp;Sharon Lionel,&nbsp;Eunice Sindhuvi,&nbsp;Alok Srivastava,&nbsp;Biju George,&nbsp;Aby Abraham","doi":"10.31547/bct-2021-020","DOIUrl":"https://doi.org/10.31547/bct-2021-020","url":null,"abstract":"<p><p>Allogeneic hematopoietic stem cell transplantation (HSCT) is a feasible treatment option for Gaucher disease (GD). Among 60 patients diagnosed with GD over 15 years (2004-2019), three children who underwent HSCT (January-November 2017) were analyzed. Two boys (cases 1 and 2) and one girl (case 3) received HSCT at 3, 7, and 10 years of age, respectively. Cases 1 and 3 received haplo-HSCT, while case 2 received HLA-identical related-donor transplantation. The CD 34 cell dose was 5-10×10⁶/kg. Neutrophil and platelet engraftment were between days +14 to +21 and days +15 to +76. Post-HSCT chimerism was a 100% donor. None of the patients developed acute or significant chronic graft versus host disease (GVHD). All patients had febrile episodes with negative blood cultures. Major post-HSCT complications included EBV-viremia and recurrent lobar pneumonia in case 1, delayed engraftment and pure red cell aplasia (PRCA) in case 2, and pericardial effusion with tamponade in case 3. At a median of 49 months post-HSCT, all patients were stable with improved growth, absent organomegaly, and had completed immunization. The median cost of treatment was $23,038.96, which is 10.7%-13% of the yearly enzyme replacement therapy (ERT) cost. In a resource-limited setting like India, ERT is a financial burden and not a sustainable option. With improved treatment outcomes, haplo-HSCT is now a possible option for almost every patient, even if no HLA-identical donor is identified.</p>","PeriodicalId":72423,"journal":{"name":"Blood cell therapy","volume":"5 3","pages":"69-74"},"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/46/07/2432-7026-5-3-0069.PMC9873422.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10584930","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}