Down syndrome-associated acute lymphoblastic leukaemia (DS-ALL) is associated with inferior outcomes compared with non-DS-ALL; however, data on haematopoietic stem cell transplantation (HSCT) in DS-ALL remain limited. We analysed nationwide data of patients aged <30 years with B-cell precursor ALL who underwent first allogeneic HSCT between 2000 and 2022 in Japan. In total, 56 patients with DS-ALL and 3873 with non-DS-ALL were identified. The incidences of neutrophil engraftment, grade II-IV acute graft-versus-host disease and chronic graft-versus-host disease were comparable between groups. The 4-year event-free survival (EFS) was lower in DS-ALL than in non-DS-ALL (40.3% vs. 55.2%), but was similar when stratified by disease status at HSCT. The 4-year EFS rates in first and second complete remission (CR) were 62.7% and 48.2% in DS-ALL and 69.5% and 56.0% in non-DS-ALL respectively. Relapse, rather than non-relapse mortality (NRM), was the leading cause of treatment failure in DS-ALL. Among patients with DS-ALL undergoing HSCT in CR1/2, myeloablative conditioning (MAC) showed a trend towards superior EFS compared with reduced-intensity conditioning (RIC), which was associated with a higher incidence of NRM. Accordingly, patients in CR1/2 who are unable to tolerate MAC are considered good candidates for emerging novel therapies rather than RIC-HSCT.

Chimeric antigen receptor (CAR) T-cell therapy is effective for treating relapsed or refractory B-cell lymphoma; however, its outcomes remain heterogeneous. We conducted a nationwide retrospective registry study in Japan to assess whether pretreatment body mass index (BMI) influences outcomes after CD19-directed CAR T-cell therapy. Among 944 patients with relapsed or refractory B-cell lymphoma treated between 2019 and 2024, BMI was categorized as Low (<18.5 kg/m²; n = 166), Normal (18.5 to 24.9 kg/m²; n = 619), or High (≥25 kg/m²; n = 159). The primary endpoint was overall survival (OS). Secondary endpoints included progression-free survival (PFS), cumulative incidence of relapse or progression (CIR), non-relapse mortality (NRM), and CAR T-cell-related toxicities. Six-month OS rates in the low, normal, and high BMI groups were 73.2%, 83.7%, and 89.3%, respectively (P < .01), and corresponding PFS was 62.3%, 75.6%, and 77.7% (P < .01). CIR was more frequent in the Low-BMI group, whereas NRM did not differ significantly across groups. In the overall cohort, Low-BMI was associated with shortened OS and PFS and higher CIR in multivariable analyses. In a complete-case sensitivity analysis restricted to patients with available disease status data at the time of infusion, these associations were attenuated after adjusting for disease status. Severe CAR T-cell-related toxicities were comparable across BMI groups. Pretreatment BMI may serve as a simple clinical marker associated with early outcomes after CAR T-cell therapy, although this association may be partly explained by disease status at infusion or unmeasured disease burden.

BACKGROUND:Pazopanib is used to treat relapsed and refractory sarcomas. Pazopanib's role in pediatric, adolescent, and young adult populations remains unestablished. METHODS:To assess pazopanib's utility, we analyzed retrospectively collected data from pediatric (0-14 years) and adolescent and young adult (15-39 years) patients diagnosed with relapsed or refractory sarcomas who received pazopanib. RESULTS:We assessed data from 21 patients (10 pediatric, 11 adolescent, and young adult). Their diagnoses included osteosarcoma (n = 11), rhabdomyosarcoma (n = 4), alveolar soft part sarcoma (n = 5), and leiomyosarcoma (n = 1). Thirteen (62%) patients presented with metastatic disease at the initial diagnosis. Patients had received a median of three prior chemotherapy regimens (range: 0-6). The median duration of pazopanib treatment was 3.5 months (range: 1-12) for pediatric patients and 4 months (range: 1-83) for adolescents and young adults. Nine patients (five adolescents and young adults) discontinued pazopanib owing to disease progression, and two discontinued owing to adverse events (pneumothorax). We observed seven cases of stable disease (four adolescents and young adults) and 12 of progressive disease (six adolescents and young adults) after ~3 months. The median survival following pazopanib initiation was 7.8, 4.8, and 12.4 months for overall, pediatric, and adolescent and young adult patients, respectively. CONCLUSIONS:In a small cohort of children and adolescent and young adult patients with heavily pretreated relapsed or refractory sarcoma, pazopanib may be a feasible option. Further research on optimal therapeutic timing and the target population for pazopanib's indication is required.

Background Asparaginase (Asp) is a key component in the treatment of children with acute lymphoblastic leukemia (ALL), particularly in the induction and consolidation phases. However, hypersensitivity and other toxicities frequently lead to premature discontinuation, thus raising concerns about its impact on long-term outcomes. A nationwide clinical trial, the ALL-B12 study, was conducted in Japan between 2012 and 2017 to investigate the effectiveness of unvalidated treatment phases in each risk group of pediatric patients aged 1–19 with B-cell precursor ALL (BCP-ALL), through randomized controlled trials and establish a safety-focused treatment framework for BCP-ALL. The main results of ALL-B12 were reported in 2024. The current study aimed to evaluate the clinical implications of L-Asp truncation in this large cohort of children with BCP-ALL treated under the ALL-B12 protocol in Japan. Methods A total of 1,804 patients were included in the full analysis set of the ALL-B12 study. Patients with NCI-SR and NCI-HR were assigned to the standard risk group (SR) and the intermediate risk group (IR), respectively. Poor steroid responders and those with high-risk genomic abnormalities were assigned to the high risk group (HR). In the current analysis, patients were categorized based on whether they completed the full L-Asp schedule (complete group) or discontinued prematurely (discontinuation group). A subset of HR patients who discontinued L-Asp received vincristine and dexamethasone pulses to compensate for the treatment reduction. For SR/IR patients no additional modification was applied when L-Asp was discontinued. Clinical characteristics and outcomes were compared between these two groups. We conducted a landmark analysis of patients who remained event-free at key treatment landmarks in each group—the start of maintenance therapy for all patients, and the date of stem cell transplantation only for HR patients who received transplantation. Multivariable analysis was performed using a Cox regression model with follow-up starting from the end of induction, treating L-Asp discontinuation as a time-dependent covariate. In Japan, Erwinia asparaginase was not approved for use and could not be used as a substitute for native L-asparaginase in case of discontinuation during the study period. Asparaginase activity levels were not measured in this study. Results Among the 1,804 patients, 142 (7.9%) discontinued L-Asp prematurely. The most frequent reason for discontinuation was acute pancreatitis (AP, n = 81, 4.5% of all patients), followed by clinical hypersensitivity (CH, n = 55, 3.0%). Patients with AP discontinued L-Asp earlier: 53 /81 experienced events in the induction phase, whereas only 5/55 patients with CH experienced events in the induction phase. The analysis of clinical characteristics suggested that the discontinuation group was older at diagnosis (median 7 years old vs. 4 years old), and was thus more likely to be classified as NCI-HR compared to the complete group. Landmark analyses revealed that the 5-year event-free survival (5yEFS) were 82.4% vs. 91.1% among SR patients (p = 0.087), 70.7% vs. 83.9% among IR patients (p = 0.054), and 75.9% vs. 82.9% among HR patients (p = 0.346) in the discontinuation and complete groups, respectively. While discontinued earlier, the EFS was not different between those with AP and CH (5yEFS, 75.9% vs. 76.3%; p = 0.882). According to the Cox regresssion analysis, the effects of L-Asp discontinuation on outcomes differed across subgroups. Those who showed a poor response to the treatment (prednisolone poor responder, day 15 bone marrow blast < 25%, or MRD < 10-4 at the end of induction or at the end of consolidation) had worse outcomes when L-Asp was discontinued. Mulitivariable analysis including clinical risk factors (age/CNS status/WBC count at diagnosis, and MRD status at the end of induction) revealed a significant association between L-Asp discontinuation and EFS (HR 1.55; 95% CI, 1.02–2.36; p = 0.041). Discussion We found that BCP-ALL patients with truncated L-Asp treatment had worse EFS than those who completed L-Asp treatment, which may be partly attributed to the older age at diagnosis in the discontinuation group. Given that the intensified L-Asp arm did not show a significant benefit in the randomized controlled study within ALL-B12, our data support the existence of an appropriate and balanced dose of asparaginase tailored to the specific needs of each risk group.

BackgroundEpigenetic dysregulation plays a central role in pediatric acute myeloid leukemia (AML), yet its clinical relevance remains underexplored. This study primarily aimed to elucidate the clinical effect of H3K27me3 and H3K4me3 status on pediatric acute myeloid leukemia. We evaluated the prognostic impact of H3K27me3 and H3K4me3 histone trimethylation, along with associated gene expression profiles, in pediatric AML.MethodsWe retrospectively analyzed 74 children with newly diagnosed non-FAB M3 and non-Down syndrome AML in a prolonged cohort in Japan. Bone marrow immunohistochemistry assessed H3K27me3 and H3K4me3 expression levels. RNA sequencing was successfully performed on sorted leukemic blasts in six representative cases, owing to limited sample availability. Chemoresistance and epigenetic modulation were evaluated in AML cell lines treated with GSK-J4, a histone demethylase inhibitor.ResultsHigh H3K27me3 expression at diagnosis was significantly associated with superior overall and event-free survival over three years (OS HR 8.0; EFS HR 5.0; both p < 0.01). H3K4me3 levels at diagnosis showed no prognostic impact. Among 14 KMT2A-rearranged cases, all six patients with high H3K27me3 achieved a long-term first remission (median follow-up: 10 years), whereas those with low expression had higher relapse rates. Transcriptomic analysis revealed upregulation of HOXA9, and HOXA-cluster genes and downregulation of ABCB1, in low H3K27me3 samples. In vitro, GSK-J4 increased H3K27me3 and suppressed HOXA9 expression in KG-1 cells, enhancing sensitivity to cytarabine.ConclusionLow H3K27me3 expression defines a poor-risk group in pediatric AML, potentially via HOXA9-driven dysregulation. H3K27me3 may serve as a prognostic biomarker and potential therapeutic target.

BackgroundPediatric and adolescent/young adult (AYA) cancer patients face profound psychological challenges, exacerbated by limited access to continuous mental health support. While conventional therapeutic interventions often follow structured protocols, the potential of generative artificial intelligence (AI) chatbots to provide continuous conversational support remains unexplored. This study evaluates the feasibility and impact of AI chatbots in alleviating psychological distress and enhancing treatment engagement in this vulnerable population.MethodsTwo age-appropriate AI chatbots, leveraging GPT-4, were developed to provide natural, empathetic conversations without structured therapeutic protocols. Five pediatric and AYA cancer patients participated in a two-week intervention, engaging with the chatbots via a messaging platform. Pre- and post-intervention anxiety and stress levels were self-reported, and usage patterns were analyzed to assess the chatbots’ effectiveness.ResultsFour out of five participants reported significant reductions in anxiety and stress levels post-intervention. Participants engaged with the chatbot every 2–3 days, with sessions lasting approximately 10 min. All participants noted improved treatment motivation, with 80% disclosing personal concerns to the chatbot they had not shared with healthcare providers. The 24/7 availability particularly benefited patients experiencing nighttime anxiety.ConclusionsThis pilot study demonstrates the potential of generative AI chatbots to complement traditional mental health services by addressing unmet psychological needs in pediatric and AYA cancer patients. The findings suggest these tools can serve as accessible, continuous support systems. Further large-scale studies are warranted to validate these promising results.

British Journal of HaematologyEarly View IMAGES IN HAEMATOLOGY Paediatric anaplastic large-cell lymphoma with cardiac tamponade Takahiro Shiwaku, Corresponding Author Takahiro Shiwaku [email protected] orcid.org/0009-0001-0900-1953 Department of Pediatrics, Okayama University Hospital, Okayama, Japan Email: [email protected]Search for more papers by this authorKosuke Tamefusa, Kosuke Tamefusa orcid.org/0000-0002-1675-7038 Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this authorHisashi Ishida, Hisashi Ishida orcid.org/0000-0002-3391-8403 Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this authorKaori Fujiwara, Kaori Fujiwara Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this authorKana Washio, Kana Washio Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this authorHirokazu Tsukahara, Hirokazu Tsukahara Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this author Takahiro Shiwaku, Corresponding Author Takahiro Shiwaku [email protected] orcid.org/0009-0001-0900-1953 Department of Pediatrics, Okayama University Hospital, Okayama, Japan Email: [email protected]Search for more papers by this authorKosuke Tamefusa, Kosuke Tamefusa orcid.org/0000-0002-1675-7038 Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this authorHisashi Ishida, Hisashi Ishida orcid.org/0000-0002-3391-8403 Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this authorKaori Fujiwara, Kaori Fujiwara Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this authorKana Washio, Kana Washio Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this authorHirokazu Tsukahara, Hirokazu Tsukahara Department of Pediatrics, Okayama University Hospital, Okayama, JapanSearch for more papers by this author First published: 12 January 2025 https://doi.org/10.1111/bjh.19980Read the full textAboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onEmailFacebookxLinkedInRedditWechat No abstract is available for this article. Early ViewOnline Version of Record before inclusion in an issue RelatedInformation

The "histone code," defined by the combinatorial patterns of post-translational modifications (PTMs) on histones, plays a pivotal role in chromatin structure and gene expression. Tools for the regioselective introduction of histone PTMs in living cells are critical for dissecting the functions of these epigenetic marks. Here, we report the design and development of three regioselective catalysts that acetylate distinct lysine residues (K43, K108, and K120) on histone H2B. Using a combination of molecular dynamics simulations of catalyst-nucleosome complexes and systematic experimental optimization of catalyst structures, we identified key design principles for achieving regioselectivity. Specifically, excluding highly reactive off-target lysine residues from the catalyst effective region (CER) while maintaining proximity to a target lysine residue proved crucial. Biochemical and cellular analyses of the catalytic histone acetylation revealed that each lysine acetylation elicited unique effects on the binding affinity and activity of nucleosome-interacting molecules, as well as on transcriptional programs and cellular phenotypes. These findings establish a framework for designing regioselective histone acetylation catalysts and advance our understanding of the regulatory mechanisms underlying histone PTMs.

This study investigated the potential of the metaverse in providing psychological support for pediatric and AYA cancer patients, with a focus on those with rare cancers. The research involved ten cancer patients and survivors from four distinct regions in Japan, who participated in metaverse sessions using customizable avatars, facilitating interactions across geographical and temporal barriers. Surveys and qualitative feedback were collected to assess the psychosocial impact of the intervention. The results demonstrated that the metaverse enabled patients to connect with peers, share experiences, and receive emotional support. The anonymity provided by avatars helped reduce appearance-related anxiety and stigma associated with cancer treatment. A case study of a 19-year-old male with spinal Ewing’s sarcoma highlighted the profound emotional relief fostered by metaverse interactions. The findings suggest that integrating virtual spaces into healthcare models can effectively address the unique needs of pediatric and AYA cancer patients, offering a transformative approach to delivering psychosocial support and fostering a global patient community. This innovative intervention has the potential to revolutionize patient care in the digital age.

The treatment efficiency and predictors of atezolizumab plus bevacizumab therapy for unresectable hepatocellular carcinoma in real-world practice have not been established. This study aimed to assess the efficacy and safety of atezolizumab plus bevacizumab and to investigate predictors of progression-free survival and overall survival. Patients with unresectable hepatocellular carcinoma treated with atezolizumab plus bevacizumab therapy in 19 hospitals were enrolled before treatment and observed prospectively. The outcomes of 222 patients in this cohort were analyzed. The objective response rate and disease control rate were 22.0% and 70.6%, respectively, whereas the median progression-free survival was 5.7 months. Independent risk factors for shortened progression-free survival were younger age (<75 years; 3.9 months vs. 8.6 months), higher number of intrahepatic tumors (≥5; 4.0 months vs. 7.9 months), macrovascular invasion (2.3 months vs. 6.7 months), and higher neutrophil-to-lymphocyte ratio (≥3.03; 3.0 months vs. 7.8 months). The median overall survival was not reached; however, independent risk factors for shortened overall survival were absence of hyperlipidemia, higher number of intrahepatic tumors (≥5), macrovascular invasion, higher α-fetoprotein level (≥400 ng/mL), worse Child-Pugh score (≥6), and higher neutrophil-to-lymphocyte ratio (≥3.03). Severe adverse events (grade ≥3) were observed in 96 patients (36.0%), with proteinuria being the most frequent. In conclusion, patients with older age, lower number of intrahepatic tumors, absent macrovascular invasion, and lower neutrophil-to-lymphocyte ratio are expected to have better progression-free survival with atezolizumab plus bevacizumab therapy for unresectable hepatocellular carcinoma.

Pediatric ALK-positive anaplastic large cell lymphoma is a rare subtype of non-Hodgkin lymphoma, and approximately 30% of patients relapse following treatment with conventional chemotherapy. Alectinib monotherapy has demonstrated excellent activity in relapsed and refractory ALCL, but its role as a maintenance therapy after hematopoietic cell transplantation is unclear. We experienced a relapse case of pediatric ALK-positive ALCL with central nervous system involvement treated with alectinib maintenance therapy following cord blood transplantation. The patient has maintained complete remission for more than 3 years after transplantation. There were no remarkable adverse effects that led to discontinuation of alectinib.

BackgroundAsparaginase is essential for treating T-cell acute lymphoblastic leukemia (T-ALL). Despite the ongoing debate on whether T-ALL and T-cell lymphoblastic lymphoma (T-LBL) are the same disease entity or two distinct diseases, patients with T-LBL often receive the same or similar treatment protocols as those with T-ALL.MethodsThe outcomes of patients with or without L-asparaginase discontinuation were retrospectively analyzed among four national protocols: Japan Association of Childhood Leukemia Study (JACLS) ALL-02 and ALL-97 for T-ALL and Japanese Pediatric Leukemia/Lymphoma Study Group ALB-NHL03 and JACLS NHL-98 for T-LBL. The hazard ratio (HR) was calculated with the Cox regression model by considering L-asparaginase discontinuation as a time-dependent variable.ResultsIn total, 199 patients with T-ALL, and 133 patients with T-LBL were included. L-asparaginase discontinuation compromised event-free survival (EFS) of T-ALL patients (ALL-02: HR 3.32, 95% confidence interval [CI] 1.40-7.90; ALL-97: HR 3.39, 95%CI 1.19-9.67). Conversely, EFS compromise was not detected among T-LBL patients (ALB-NHL03: HR 1.39, 95%CI 0.41-4.68; NHL-98: HR 0.92, 95%CI 0.11-7.60).ConclusionThe effects of L-asparaginase discontinuation differed between T-ALL and T-LBL. We assume that the differential impact results from (1) the inherent differential response to L-asparaginase between them and/or (2) a less stringent assessment of early treatment response in T-LBL than in T-ALL. Given the poor salvage rate of refractory or relapsed T-ALL and T-LBL, optimization of the frontline therapy is critical, and the current study provides a new suggestion for further treatment modifications. However, larger studies in contemporary intensified treatment protocols are required. The outcomes of patients with or without L-asparaginase discontinuation were retrospectively analyzed among four national protocols, two with T-cell acute lymphoblastic leukemia (T-ALL) and two with T-cell lymphoblastic lymphoma (T-LBL). The effects of L-asparaginase discontinuation differed between T-ALL and T-LBL, and given a poor salvage rate of refractory or relapsed T-ALL and T-LBL, optimization of the frontline therapy is critical, and the current study provided a new suggestion for further treatment modifications.image

Objective: This study primarily focused on the diagnostic interval (DI), defined as the duration from the onset of leukemic symptoms to diagnosis. We investigated whether a prolonged DI is associated with the outcomes of pediatric leukemia.Methods: We retrospectively collected data of children with newly diagnosed pediatric leukemia at Okayama University Hospital from January 2007 to December 2022. Survival analyses were conducted using Kaplan-Meier methods, and an unadjusted analysis to compare differences in survival was performed using the log-rank test.Results: In total, 103 children with leukemia were included in the analysis. The median DI was 20 days (interquartile range, 9.5-33.5 days). A prolonged DI (>= 30 days) demonstrated no association with either 5-year event-free survival (70.1% for <30 days and 68.3% for >= 30 days, p = .99, log-rank test) or overall survival (84.7% for <30 days and 89.4% for >= 30 days, p = .85, log-rank test).Conclusions: A prolonged DI was not associated with the survival of children with leukemia. If a precise classification of leukemia biology is provided for pediatric patients, a prolonged DI may have little impact on the prognosis of these patients.

Cytomegalovirus (CMV) infection is a major complication of hematopoietic stem cell transplantation (HSCT). Previous studies in adults demonstrated that letermovir prophylaxis for 100 d after HSCT reduces the occurrence of CMV infection; however, studies in children are limited. In this study, we aimed to examine the incidence of CMV infection in children who underwent allogeneic HSCT with prophylactic letermovir therapy. A single-center retrospective study was conducted among patients aged ≤17 who underwent allogeneic HSCT. We compared the cumulative incidence of CMV infection, mainly monitored by pp65-antigenemia, after HSCT between patients with and without letermovir prophylaxis (10-12 or 5-6 mg/kg/d when co-administered with cyclosporine) using Gray's test. We analyzed 79 patients with a median follow-up period of 126 d. The median age of these patients was 8.3 years (Interquartile range, 3.7-12.4). Prophylactic letermovir was used in 25 patients. Twenty-five patients developed CMV infection, and the cumulative incidence was 38.9% (95% confidence intervals, 25.0-52.5). The cumulative incidence of CMV infection was not significantly different between the letermovir and no-letermovir groups (33.1 vs. 36.6%, p = 0.228). Meanwhile, the cumulative incidence of CMV infection up to 100 d following HSCT was significantly lower in the letermovir group than in the no-letermovir group (8.0 vs. 32.8%, p = 0.026). Most patients experienced no noticeable adverse effects associated with letermovir; however, one patient discontinued letermovir because of nausea and anorexia. In conclusion, the results of this study suggest that letermovir prophylaxis against CMV infection may be effective in children without severe adverse effects.

60歳女性．肝細胞癌多発肺転移に対してアテゾリズマブ・ベバシズマブ併用療法を開始した14日後から四肢末梢の感覚障害と四肢の筋力低下が出現した．脳脊髄液検査では蛋白および細胞数の上昇を認め，血清中のGalNAc-GD1a IgG抗体とGal-C IgG抗体が陽性であった．神経伝導速度検査で脱髄型末梢神経障害を認め，アテゾリズマブ関連の薬物性ギラン・バレー症候群と診断した．大量免疫グロブリン療法にて筋力低下は軽快した．今回，肝細胞癌に対して使用した免疫チェックポイント阻害薬として免疫関連副作用でギラン・バレー症候群を呈し，早期治療介入により症状改善を得た症例を経験したため報告する．

Survival rates of patients with Philadelphia chromosome-positive ALL (Ph+ALL) have improved considerably with the introduction of tyrosine kinase inhibitors (TKI); however, hematopoietic stem cell transplantation (HSCT) continues to play an important role. Reduced-intensity conditioning (RIC) regimens have been widely applied particularly for older patients, but their validity for children and adolescents with Ph+ALL has not been investigated. In this study, data from patients receiving HSCT for de novo Ph+ALL in first or second remission at ages younger than 25 years and with a history of pre-HSCT TKI therapy were retrospectively collected through the nationwide registry in Japan. In 265 patients who received myeloablative conditioning (MAC) and 33 patients receiving RIC, 5-year leukemia-free survival (LFS) rates were 67.3% and 79.8%, respectively (p = 0.142). Multivariate analysis of LFS, focusing on patients with good performance status, identified RIC as a significant prognostic factor for LFS (hazard ratio 0.32, p = 0.032), as well as older age, higher leukocyte count at diagnosis, and disease with additional chromosomal abnormalities. These trends were similar when we focused on patients who received prophylactic post-HSCT TKI treatment, as 5-year LFS was 81.0% for MAC and 84.4% for RIC (p = 0.748). In summary, HSCT with RIC regimen showed at least comparable LFS to HSCT with MAC regimen, and RIC was an independent favorable prognostic factor on multivariate analysis adjusting potential prognostic factors. While patient numbers were limited, our data suggest that RIC may be safely applied in this group, particularly combined with prophylactic post-HSCT TKI maintenance therapy.