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A Multistakeholder Qualitative Analysis of Barriers to Autologous Stem Cell Transplantation for Multiple Myeloma 多发性骨髓瘤自体干细胞移植障碍的多利益相关者定性分析。
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.07.002
James Fan Wu , Idayat M. Akinola , Anita D’Souza , Rachel Cusatis

Purpose

We investigated autologous stem cell transplantation (ASCT) barriers in a single-center qualitative study by interviewing patients with multiple myeloma (MM), transplant physicians (TPs), and referring physicians (RPs).

Methods

Patients with MM and referred for ASCT evaluation between January 01, 2021 and December 31, 2022, TPs from 2 Wisconsin transplant centers, and RPs from rural and urban Wisconsin were recruited. Interviews were conducted using a semi-structured interview guide focused on ASCT barriers. Transcripts were double-coded and reviewed. An initial codebook with prespecified themes based on the interview guide was developed and iteratively revised.

Results

Eighteen patients, 5 TPs, and 4 RPs were interviewed. Four major ASCT barriers themes were identified across all stakeholders: caregiver support, patient concerns and knowledge, financial toxicity, and logistics. Unique themes included social support by patients and ASCT referral by physicians. Lacking caregiver support was more common among single, childless, and socially/geographically isolated patients. Patients were most concerned about side effects, long hospital stays, and quality of life. Patients used online websites and support groups to gain knowledge and support. While most patients experienced financial toxicity, care costs were rarely discussed. Distance from transplant center, transportation, and care coordination were barriers. Referrals from community oncologists are a major barrier. Patients never seen at a transplant center may have the most barriers.

Conclusion

This qualitative study of both patients and physicians investigating barriers to access to ASCT in MM, provides a much-needed multistakeholder perspective. Interventional strategies targeting key barriers will be critical in improving ASCT utilization in MM.
目的:我们通过采访多发性骨髓瘤(MM)患者、移植医生(tp)和转诊医生(rp),在一项单中心定性研究中调查了自体干细胞移植(ASCT)障碍。方法:招募2021年1月1日至2022年12月31日期间接受ASCT评估的MM患者,来自威斯康星州2个移植中心的TPs,以及威斯康星州农村和城市的RPs。访谈采用半结构化访谈指南,重点关注ASCT障碍。记录被双重编码和审查。根据采访指南编写了一份带有预先指定主题的初始代码本,并进行了反复修订。结果:访谈18例患者,5例TPs, 4例RPs。在所有利益相关者中确定了四个主要的ASCT障碍主题:护理人员支持、患者关注和知识、财务毒性和物流。独特的主题包括患者的社会支持和医生的ASCT转诊。缺乏照顾者支持在单身、无子女和社会/地理隔离的患者中更为常见。患者最关心的是副作用、住院时间和生活质量。患者利用在线网站和支持小组获得知识和支持。虽然大多数患者都经历了经济损失,但很少讨论护理费用。与移植中心的距离、交通和护理协调是障碍。来自社区肿瘤学家的转诊是一个主要障碍。从未在移植中心见过的患者可能面临最大的障碍。结论:这项定性研究调查了MM患者和医生获得ASCT的障碍,提供了急需的多利益相关者视角。针对关键障碍的干预策略对于提高急性粒细胞白血病患者ASCT的利用至关重要。
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引用次数: 0
Variables Associated With the Risk of Severe Mucositis During Autologous Transplantation in Multiple Myeloma Patients 多发性骨髓瘤患者自体移植时发生严重粘膜炎风险的相关变量
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.08.018
Irene Francés Alexandre , Ana Gea Peña , Ana Tobalina García , Juan José Domínguez-García , Elena Alejo Alonso , Ana Pilar González-Rodríguez , Irene Padilla , Maialen Sirvent Auzmendi , Ana Lobo Olmedo , Ana María Vale López , Irene Castaño Caballero , Evelyn Valencia , Paula Melero Valentín , Ianire Etxeguren Urkixo , María de las Mercedes Luque Romero , Javier Larreina Pérez , Borja Puertas Martínez , Fernando Escalante Barrigón , María Oviedo Madrid , Julia Bannatyne Undabeitia , Enrique María Ocio

Introduction

Autologous stem cell transplantation (ASCT) with melphalan 200 mg/m² remains a standard treatment for multiple myeloma (MM). We analyzed the incidence and risk factors associated with the development of mucositis in 1,268 MM patients who underwent ASCT between 2017 and 2022 across 13 national centers.

Results

Median age was 60 years (24-77), and 44% were female. Induction therapy included chemotherapy or cyclophosphamide (principally VCD) in 32%, and 21% had creatinine > 1 mg/dL. Melphalan was administered in a single day (Mel1) in 52% and over 2 days (Mel2) in 48%. Prophylaxis measures included cryotherapy (75%), G-CSF (78%) and antibiotics (37%). Mucositis occurred in 84% of patients, with 26% experiencing severe cases (WHO grade III-IV), which were significantly associated with increased intravenous opioids use, parenteral nutrition, and febrile episodes. Severe mucositis was more common in women (32% vs. 22%), patients over 60 (53% vs. 60%), chemotherapy induction (37% vs. 20%), creatinine > 1 mg/dL (34% vs. 24%), those not receiving G-CSF (35% vs. 24%) or antibiotic (36% vs. 16%). It was also more common with single day melphalan (29% vs. 24%). In the multivariable analysis, all factors except chemotherapy-based induction were identified as independent predictors of severe mucositis.

Conclusion

In our population, an increased incidence of severe mucositis was observed in nonmodifiable factors such as sex and renal function. However, the implementation of modifiable factors, including the use of prophylactic G-CSF or antibiotics and administering melphalan over 2 days could diminish the severity of this complication.
自体干细胞移植(ASCT)加200 mg/m²melphalan仍然是多发性骨髓瘤(MM)的标准治疗方法。我们分析了13个国家中心2017年至2022年间接受ASCT的1268名MM患者的粘膜炎发病率和相关危险因素。结果:中位年龄为60岁(24-77岁),女性占44%。诱导治疗包括32%的化疗或环磷酰胺(主要是VCD), 21%的肌酐为1 mg/dL。52%的患者在一天内(Mel1)给药,48%的患者在2天内(Mel2)给药。预防措施包括冷冻治疗(75%)、G-CSF(78%)和抗生素(37%)。84%的患者发生粘膜炎,其中26%出现严重病例(世卫组织III-IV级),这与静脉注射阿片类药物使用增加、肠外营养和发热发作显著相关。严重粘膜炎在女性(32%比22%)、60岁以上患者(53%比60%)、化疗诱导(37%比20%)、肌酐bbb10 1 mg/dL(34%比24%)、未接受G-CSF(35%比24%)或抗生素(36%比16%)的患者中更为常见。服用一天的美法仑也更常见(29%对24%)。在多变量分析中,除了基于化疗的诱导外,所有因素都被确定为严重粘膜炎的独立预测因素。结论:在我们的人群中,性别和肾功能等不可改变的因素增加了严重粘膜炎的发病率。然而,实施可改变的因素,包括使用预防性G-CSF或抗生素,并给予2天以上的美法兰,可以减少这种并发症的严重程度。
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引用次数: 0
Association and Prognostic Significance of Comorbidities in Individuals With MGUS MGUS患者合并症的相关性及预后意义。
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.08.019
Sawyer Bawek , Nour Nassour , Malak Alharbi , Alan Hutson , Sarah Parker , Joseph D. Tario , Hamza Hassan , Scott I. Abrams , Philip L. McCarthy , Jens Hillengass , Janine M. Joseph

Introduction

Monoclonal gammopathy of undetermined significance (MGUS) is a benign precursor of multiple myeloma, but little is known about the role of comorbidities in progression.

Materials and Methods

A retrospective review of medical records of patients with MGUS (n = 150) or a negative MGUS workup (n = 96) seen at a comprehensive cancer center between 2007 and 2023 was performed. Charlson Comorbidity Index (CCI) scores, descriptive characteristics, and overall (OS) and progression-free (PFS) survival were abstracted. Group differences (MGUS vs negative) in CCI and other characteristics were tested with Student’s t-tests and Pearson’s chi-square tests. Univariate and multivariate Cox proportional hazard models were used to model the association between CCI and OS and PFS in patients with MGUS.

Results

Patients with MGUS had a higher mean CCI (P = .01) and prevalence of diabetes mellitus with end-organ damage (P < .05) and solid tumors (P = .01) than patients with a negative MGUS workup. Each additional CCI point was associated with shorter OS (HR 1.50, P< .01) and PFS (HR 1.25, P < .01). Results were not confounded by age, body mass index, or sex.

Conclusions

Our study suggests that comorbidities are relevant to risk of MGUS and that prevention and management of comorbidities in patients with MGUS is indicated and may be associated with improved outcomes.
摘要:意义未确定的单克隆γ病(MGUS)是多发性骨髓瘤的良性前体,但对合并症在进展中的作用知之甚少。材料和方法:回顾性分析2007年至2023年在一家综合癌症中心就诊的MGUS患者(n = 150)或MGUS阴性患者(n = 96)的医疗记录。对Charlson共病指数(CCI)评分、描述性特征、总生存期(OS)和无进展生存期(PFS)进行抽象。CCI及其他特征的组间差异(MGUS vs阴性)采用Student’st检验和Pearson’s卡方检验。采用单因素和多因素Cox比例风险模型对MGUS患者CCI与OS和PFS之间的关系进行建模。结果:与MGUS阴性患者相比,MGUS患者的CCI平均值(P = 0.01)、糖尿病伴终末器官损害(P < 0.05)和实体瘤患病率(P = 0.01)均较高。每增加一个CCI点,OS (HR 1.50, P< 0.01)和PFS (HR 1.25, P< 0.01)均缩短。结果不受年龄、体重指数或性别的影响。结论:我们的研究表明,合并症与MGUS的风险相关,MGUS患者的合并症的预防和管理是必要的,可能与预后的改善有关。
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引用次数: 0
Survival Outcomes in Patients With Chronic Lymphocytic Leukemia: A SEER Analysis 慢性淋巴细胞白血病患者的生存结局:一项SEER分析
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.07.012
Shivani Modi , Srinishant Rajarajan , Hardik Jain , Claudia Dourado

Survival Outcomes

Chronic lymphocytic leukemia (CLL) is the most prevalent adult leukemia. CLL treatment has evolved dramatically from alkylator-based regimens to chemoimmunotherapy and targeted therapies. This study evaluated real-world survival outcomes across treatment eras using population-based data, focusing on patients who received active systemic therapy to assess treatment effectiveness rather than natural disease progression.

Methods

We conducted a retrospective cohort study using surveillance, epidemiology, and end results (SEER) data from 1995 to 2020, restricting analysis to CLL patients who received active systemic treatment. Patients were stratified into three treatment eras based on treatment initiation date: Pre-novel therapy era (1995-2004, alkylator-based regimens), Early novel therapy era (2005-2014, chemoimmunotherapy with anti-CD20 monoclonal antibodies), and modern novel therapy era (2015-2020, targeted therapies including BTK inhibitors and BCL-2 inhibitors). Era boundaries accounted for the lag between regulatory approval and community adoption. Overall survival was calculated from treatment initiation date, with era assignment based on first-line therapy to maintain temporal consistency.

Results

Among 49,056 CLL patients across three treatment eras, median overall survival improved significantly from 3.5 years (95% CI, 3.2-3.8) in the pre-novel therapy era (1995-2004) to 5.2 years (95% CI, 4.9-5.6) in the early novel therapy era (2005-2014), and 7.8 years (95% CI, 6.9-8.7) in the modern novel therapy era (2015-2020). Multivariable Cox regression analysis demonstrated progressive reductions in mortality risk, with adjusted hazard ratios of 0.75 (95% CI, 0.68-0.82, P < .001) for the Early Novel era and 0.45 (95% CI, 0.38-0.53, P < .001) for the Modern era compared to the Pre-Novel era. Complete response rates increased from 15% to20% in the Pre-Novel era to over 60% in the Modern era with targeted therapies.

Conclusions

This analysis demonstrates significant improvements in survival outcomes for CLL patients across treatment eras, with the most pronounced benefits observed in the modern targeted therapy era. By focusing on treated patients and calculating survival from treatment initiation, we isolated the impact of therapeutic advances from disease biology and patient selection factors. However, the COVID-19 pandemic's disproportionate impact on immunocompromised CLL patients, particularly affecting the modern therapy era, represents an important limitation that may underestimate the true survival benefits of contemporary targeted therapies. These findings support the continued development and adoption of novel therapeutic strategies in CLL management.
生存结果:慢性淋巴细胞白血病(CLL)是最常见的成人白血病。CLL治疗已经从基于烷基化剂的方案发展到化学免疫治疗和靶向治疗。本研究使用基于人群的数据评估了不同治疗时期的真实生存结果,重点关注接受主动全身治疗的患者,以评估治疗效果,而不是自然疾病进展。方法:我们使用1995年至2020年的监测、流行病学和最终结果(SEER)数据进行了一项回顾性队列研究,将分析限制在接受积极全身治疗的CLL患者。根据治疗开始日期,将患者分为三个治疗时代:新疗法前时代(1995-2004年,以烷基化剂为基础的方案),早期新疗法时代(2005-2014年,抗cd20单克隆抗体的化学免疫治疗)和现代新疗法时代(2015-2020年,包括BTK抑制剂和BCL-2抑制剂的靶向治疗)。时代界限解释了监管批准和社区采用之间的滞后。总生存期从治疗开始日期计算,时间分配基于一线治疗以保持时间一致性。结果:在三个治疗时代的49056名CLL患者中,中位总生存期从新疗法前时代(1995-2004)的3.5年(95% CI, 3.2-3.8)显著提高到早期新疗法时代(2005-2014)的5.2年(95% CI, 4.9-5.6),以及现代新疗法时代(2015-2020)的7.8年(95% CI, 6.9-8.7)。多变量Cox回归分析显示,与前小说时代相比,早期小说时代的校正风险比为0.75 (95% CI, 0.68-0.82, P < .001),现代小说时代的校正风险比为0.45 (95% CI, 0.38-0.53, P < .001)。通过靶向治疗,完全缓解率从前小说时代的15% - 20%增加到现代时代的60%以上。结论:该分析表明,CLL患者在不同治疗时期的生存结果有显著改善,在现代靶向治疗时期观察到最明显的益处。通过关注治疗患者并从治疗开始计算生存率,我们将治疗进展的影响从疾病生物学和患者选择因素中分离出来。然而,COVID-19大流行对免疫功能低下的CLL患者的不成比例的影响,特别是对现代治疗时代的影响,代表了一个重要的限制,可能低估了当代靶向治疗的真正生存益处。这些发现支持在CLL治疗中持续发展和采用新的治疗策略。
{"title":"Survival Outcomes in Patients With Chronic Lymphocytic Leukemia: A SEER Analysis","authors":"Shivani Modi ,&nbsp;Srinishant Rajarajan ,&nbsp;Hardik Jain ,&nbsp;Claudia Dourado","doi":"10.1016/j.clml.2025.07.012","DOIUrl":"10.1016/j.clml.2025.07.012","url":null,"abstract":"<div><h3>Survival Outcomes</h3><div>Chronic lymphocytic leukemia (CLL) is the most prevalent adult leukemia. CLL treatment has evolved dramatically from alkylator-based regimens to chemoimmunotherapy and targeted therapies. This study evaluated real-world survival outcomes across treatment eras using population-based data, focusing on patients who received active systemic therapy to assess treatment effectiveness rather than natural disease progression.</div></div><div><h3>Methods</h3><div>We conducted a retrospective cohort study using surveillance, epidemiology, and end results (SEER) data from 1995 to 2020, restricting analysis to CLL patients who received active systemic treatment. Patients were stratified into three treatment eras based on treatment initiation date: Pre-novel therapy era (1995-2004, alkylator-based regimens), Early novel therapy era (2005-2014, chemoimmunotherapy with anti-CD20 monoclonal antibodies), and modern novel therapy era (2015-2020, targeted therapies including BTK inhibitors and BCL-2 inhibitors). Era boundaries accounted for the lag between regulatory approval and community adoption. Overall survival was calculated from treatment initiation date, with era assignment based on first-line therapy to maintain temporal consistency.</div></div><div><h3>Results</h3><div>Among 49,056 CLL patients across three treatment eras, median overall survival improved significantly from 3.5 years (95% CI, 3.2-3.8) in the pre-novel therapy era (1995-2004) to 5.2 years (95% CI, 4.9-5.6) in the early novel therapy era (2005-2014), and 7.8 years (95% CI, 6.9-8.7) in the modern novel therapy era (2015-2020). Multivariable Cox regression analysis demonstrated progressive reductions in mortality risk, with adjusted hazard ratios of 0.75 (95% CI, 0.68-0.82, <em>P</em> &lt; .001) for the Early Novel era and 0.45 (95% CI, 0.38-0.53, <em>P</em> &lt; .001) for the Modern era compared to the Pre-Novel era. Complete response rates increased from 15% to20% in the Pre-Novel era to over 60% in the Modern era with targeted therapies.</div></div><div><h3>Conclusions</h3><div>This analysis demonstrates significant improvements in survival outcomes for CLL patients across treatment eras, with the most pronounced benefits observed in the modern targeted therapy era. By focusing on treated patients and calculating survival from treatment initiation, we isolated the impact of therapeutic advances from disease biology and patient selection factors. However, the COVID-19 pandemic's disproportionate impact on immunocompromised CLL patients, particularly affecting the modern therapy era, represents an important limitation that may underestimate the true survival benefits of contemporary targeted therapies. These findings support the continued development and adoption of novel therapeutic strategies in CLL management.</div></div>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":"26 1","pages":"Pages 42-48"},"PeriodicalIF":2.7,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144944924","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Management of Chronic Myeloid Leukemia During Pregnancy: Review of Evidence and Treatment Algorithm 妊娠期慢性髓性白血病的管理:证据和治疗算法的回顾。
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.07.017
Fadi G. Haddad, Jennifer Croden, Koji Sasaki, Ghayas C. Issa, Naveen Pemmaraju, Hagop Kantarjian, Elias Jabbour
Philadelphia chromosome-positive chronic myeloid leukemia (CML) during pregnancy is rare, with an annual incidence of 1 per 100,000 pregnancies. Managing CML in pregnancy is challenging due to concerns about the teratogenicity of the BCR::ABL1 tyrosine kinase inhibitors (TKIs). While some pregnant patients with chronic-phase CML may be managed with close monitoring and no therapy, others, particularly those diagnosed in the first trimester, may require treatment to prevent disease progression and maternal complications. Imatinib has been associated with an increased risk of congenital malformations when used in the first trimester; however, growing evidence support its safety later in the second and third trimesters. Similar to imatinib, nilotinib has limited placental transfer with no congenital malformations reported to date in women who received it later in pregnancy. Dasatinib, however, is contraindicated due to its high association with congenital abnormalities and hydrops fetalis. Ponatinib exposure has been linked to a higher incidence of Hirschsprung’s disease and should be avoided in pregnancy. Although no congenital malformations have been reported with bosutinib or asciminib, data remains limited, and their use is not recommended. Interferon-α is a safer alternative during pregnancy but is slower in achieving cytoreduction, and less effective compared to TKIs. Hydroxyurea, while not teratogenic, may increase the risk of miscarriage and low birth weight. If a male patient is receiving TKI therapy, the female partner can safely become pregnant without an increased risk for the fetus. Shared decision-making and thorough counseling on the risks and benefits of different treatment options are essential. This review examines the potential fetotoxicity of CML therapies, focusing on TKIs and alternative treatments for pregnant women.
妊娠期费城染色体阳性慢性髓性白血病(CML)是罕见的,年发病率为10万分之一。由于担心BCR::ABL1酪氨酸激酶抑制剂(TKIs)的致畸性,妊娠期CML的治疗具有挑战性。虽然一些患有慢性粒细胞白血病的妊娠患者可以通过密切监测而不进行治疗,但其他患者,特别是在妊娠早期诊断出的患者,可能需要治疗以防止疾病进展和产妇并发症。伊马替尼与妊娠早期使用的先天性畸形风险增加有关;然而,越来越多的证据支持其在妊娠中期和晚期的安全性。与伊马替尼类似,尼洛替尼的胎盘移植有限,迄今为止在怀孕后期接受尼洛替尼的妇女中没有先天性畸形的报道。然而,达沙替尼是禁忌症,因为它与先天性异常和胎儿水肿高度相关。波纳替尼暴露与Hirschsprung病的高发病率有关,在怀孕期间应避免使用。虽然博舒替尼或阿西米尼没有先天性畸形的报道,但数据仍然有限,不建议使用它们。干扰素-α在妊娠期间是一种更安全的选择,但在实现细胞减少方面较慢,与TKIs相比效果较差。羟基脲虽然没有致畸性,但可能会增加流产和低出生体重的风险。如果男性患者正在接受TKI治疗,女性伴侣可以安全地怀孕,而不会增加胎儿的风险。对不同治疗方案的风险和益处进行共同决策和全面咨询是必不可少的。这篇综述探讨了CML治疗的潜在胎儿毒性,重点是TKIs和孕妇的替代治疗。
{"title":"Management of Chronic Myeloid Leukemia During Pregnancy: Review of Evidence and Treatment Algorithm","authors":"Fadi G. Haddad,&nbsp;Jennifer Croden,&nbsp;Koji Sasaki,&nbsp;Ghayas C. Issa,&nbsp;Naveen Pemmaraju,&nbsp;Hagop Kantarjian,&nbsp;Elias Jabbour","doi":"10.1016/j.clml.2025.07.017","DOIUrl":"10.1016/j.clml.2025.07.017","url":null,"abstract":"<div><div>Philadelphia chromosome-positive chronic myeloid leukemia (CML) during pregnancy is rare, with an annual incidence of 1 per 100,000 pregnancies. Managing CML in pregnancy is challenging due to concerns about the teratogenicity of the BCR::ABL1 tyrosine kinase inhibitors (TKIs). While some pregnant patients with chronic-phase CML may be managed with close monitoring and no therapy, others, particularly those diagnosed in the first trimester, may require treatment to prevent disease progression and maternal complications. Imatinib has been associated with an increased risk of congenital malformations when used in the first trimester; however, growing evidence support its safety later in the second and third trimesters. Similar to imatinib, nilotinib has limited placental transfer with no congenital malformations reported to date in women who received it later in pregnancy. Dasatinib, however, is contraindicated due to its high association with congenital abnormalities and hydrops fetalis. Ponatinib exposure has been linked to a higher incidence of Hirschsprung’s disease and should be avoided in pregnancy. Although no congenital malformations have been reported with bosutinib or asciminib, data remains limited, and their use is not recommended. Interferon-α is a safer alternative during pregnancy but is slower in achieving cytoreduction, and less effective compared to TKIs. Hydroxyurea, while not teratogenic, may increase the risk of miscarriage and low birth weight. If a male patient is receiving TKI therapy, the female partner can safely become pregnant without an increased risk for the fetus. Shared decision-making and thorough counseling on the risks and benefits of different treatment options are essential. This review examines the potential fetotoxicity of CML therapies, focusing on TKIs and alternative treatments for pregnant women.</div></div>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":"26 1","pages":"Pages 19-25"},"PeriodicalIF":2.7,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144944910","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Dermatologic Adverse Events Following CD19-Directed CAR T-Cell Therapy in Hematologic Malignancies cd19靶向CAR - t细胞治疗血液恶性肿瘤后皮肤不良事件。
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.09.012
Ryan Storgard , Rachel E. Reingold , Rose Parisi , Stephen W. Dusza , Sarah J. Noor , Jae H. Park , Craig S. Sauter , Kevin J. Curran , M. Lia Palomba , Gunjan Shah , Roni Shouval , Miguel-Angel Perales , Michael Scordo , Alina Markova

Background

CD19 CAR T-cell therapy is a significant advance in B-NHL and ALL. This study describes the incidence, onset, and factors of dermatologic adverse events (dAE) post-therapy.

Methods

A retrospective analysis at Memorial Sloan Kettering Cancer Center (4/2013-8/2020) identified 193 patients undergoing CD19 CAR T-cell therapy. We aimed to characterize dAEs post CAR T-cell therapy including the 100-day cumulative incidence, time to dAE onset, and associations with cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS).

Results

Eighty-two patients experienced 94 dAEs within the first 100 days (incidence: 0.42 (95% CI: 0.36-0.51) post CAR T-cell therapy. Common dAEs were rash (30.9%, n = 29) including maculopapular rash, inflammatory papules, and local erythema; infection (23.4%, n = 22) including cellulitis and folliculitis; and xerosis (16.0%, n = 15). Specific early onset dAEs included rash and chemotherapy-related events, eg, alopecia, mucositis (median 12- and 17-days postinfusion, respectively). Thrombocytopenic purpura and xerosis presented later (median 22-and 25-days). CRS and dAEs occurred in 33.5% of patients, with CRS preceding dAEs in 86% of cases. Among 15% with ICANS and dAEs, ICANS was antecedent in 67%.

Conclusion

dAEs following CAR T-cell therapy are common but mostly low-grade and often manifest within the initial month postinfusion.
背景:CD19 CAR - t细胞治疗是治疗B-NHL和ALL的重要进展。本研究描述了治疗后皮肤不良事件(dAE)的发生率、发病和因素。方法:对纪念斯隆-凯特琳癌症中心(2013年4月- 2020年8月)193例接受CD19 CAR - t细胞治疗的患者进行回顾性分析。我们的目的是表征CAR - t细胞治疗后dAEs的特征,包括100天累积发病率、dAE发病时间以及与细胞因子释放综合征(CRS)和免疫效应细胞相关神经毒性综合征(ICANS)的相关性。结果:82例患者在CAR -t细胞治疗后的前100天内经历了94次dAEs(发生率:0.42 (95% CI: 0.36-0.51))。常见的dAEs为皮疹(30.9%,n = 29),包括斑疹丘疹、炎性丘疹和局部红斑;感染(23.4%,n = 22)包括蜂窝织炎和毛囊炎;干枯病(16.0%,n = 15)。特异性早发性dAEs包括皮疹和化疗相关事件,如脱发、粘膜炎(中位分别为输注后12天和17天)。血小板减少性紫癜和干燥症出现较晚(中位22天和25天)。33.5%的患者发生CRS和dAEs, 86%的患者CRS先于dAEs。在15%的ICANS和dAEs患者中,有67%的人有ICANS。结论:CAR - t细胞治疗后的dAEs是常见的,但大多是低级别的,通常在输注后的第一个月内出现。
{"title":"Dermatologic Adverse Events Following CD19-Directed CAR T-Cell Therapy in Hematologic Malignancies","authors":"Ryan Storgard ,&nbsp;Rachel E. Reingold ,&nbsp;Rose Parisi ,&nbsp;Stephen W. Dusza ,&nbsp;Sarah J. Noor ,&nbsp;Jae H. Park ,&nbsp;Craig S. Sauter ,&nbsp;Kevin J. Curran ,&nbsp;M. Lia Palomba ,&nbsp;Gunjan Shah ,&nbsp;Roni Shouval ,&nbsp;Miguel-Angel Perales ,&nbsp;Michael Scordo ,&nbsp;Alina Markova","doi":"10.1016/j.clml.2025.09.012","DOIUrl":"10.1016/j.clml.2025.09.012","url":null,"abstract":"<div><h3>Background</h3><div>CD19 CAR T-cell therapy is a significant advance in B-NHL and ALL. This study describes the incidence, onset, and factors of dermatologic adverse events (dAE) post-therapy.</div></div><div><h3>Methods</h3><div>A retrospective analysis at Memorial Sloan Kettering Cancer Center (4/2013-8/2020) identified 193 patients undergoing CD19 CAR T-cell therapy. We aimed to characterize dAEs post CAR T-cell therapy including the 100-day cumulative incidence, time to dAE onset, and associations with cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS).</div></div><div><h3>Results</h3><div>Eighty-two patients experienced 94 dAEs within the first 100 days (incidence: 0.42 (95% CI: 0.36-0.51) post CAR T-cell therapy. Common dAEs were rash (30.9%, <em>n</em> = 29) including maculopapular rash, inflammatory papules, and local erythema; infection (23.4%, <em>n</em> = 22) including cellulitis and folliculitis; and xerosis (16.0%, <em>n</em> = 15). Specific early onset dAEs included rash and chemotherapy-related events, eg, alopecia, mucositis (median 12- and 17-days postinfusion, respectively). Thrombocytopenic purpura and xerosis presented later (median 22-and 25-days). CRS and dAEs occurred in 33.5% of patients, with CRS preceding dAEs in 86% of cases. Among 15% with ICANS and dAEs, ICANS was antecedent in 67%.</div></div><div><h3>Conclusion</h3><div>dAEs following CAR T-cell therapy are common but mostly low-grade and often manifest within the initial month postinfusion.</div></div>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":"26 1","pages":"Pages e120-e126"},"PeriodicalIF":2.7,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145318310","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Older Patients Aged 65 Years and Above With Myelofibrosis 同种异体造血干细胞移植治疗65岁及以上老年骨髓纤维化患者的疗效。
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.08.012
Moazzam Shahzad , Muhammad Kashif Amin , Sohaib Irfan , Rania Ahsan , Hana Qasim , Muhammad Jawad Javed , Matthew McGuirk , Sibgha Gull Chaudhary , Iqra Anwar , Abdulraheem Yacoub , Anurag K. Singh , Mehdi Hamadani , Joseph P. Mcguirk , Muhammad Umair Mushtaq

Background

Allogeneic hematopoietic stem cell transplantation (allo-HCT) remains the only curative option for myelofibrosis (MF) but is often underutilized in patients aged ≥ 65 due to concerns about treatment-related toxicity.

Methods

We conducted a retrospective analysis of chronic-phase MF allo-HCT recipients using the publicly available CIBMTR P-5640 dataset (2008-2019). Key endpoints included overall survival (OS), disease-free survival (DFS), relapse, nonrelapse mortality (NRM), and graft-vs-host disease (GVHD)-related outcomes. Hazard ratios (HR) and 95% confidence intervals (CI) were calculated using Cox regression models. Statistical analysis was performed using R (v4.3.2), with statistical significance defined as P < .05.

Results

We included 856 MF allo-HCT recipients (aged ≥ 65: n = 259, aged < 65: n = 597). The 2-year OS, DFS, and GVHD-free, relapse-free survival (GRFS) for patients ≥ 65 years were 62%, 33%, and 12%, respectively. Rates of relapse, NRM, grade II–IV acute GVHD, and chronic GVHD were 43%, 27%, 46%, and 42%, respectively. In multivariable analysis, age ≥ 65 years was independently associated with inferior DFS (HR 1.26, 95% CI 1.03-1.55, P = .025); however, there was no impact on other post-transplant outcomes. Independent predictors of worse outcomes in older patients included splenic radiation and ATG use, secondary MF, higher comorbidities (HCT-CI ≥ 3), mismatched donor type, and bone marrow graft.

Conclusions

In myelofibrosis patients aged 65 years and above undergoing allogeneic HCT, a favorable 2-year overall survival of 62% was noted with an inferior DFS compared to younger patients, while other post-transplant outcomes remained comparable. These findings support allo-HCT as a viable option in selected elderly MF patients using tailored transplant strategies.
背景:同种异体造血干细胞移植(Allogeneic hematopoietic stem cell transplantation, allo-HCT)仍然是治疗骨髓纤维化(MF)的唯一选择,但由于担心治疗相关的毒性,在年龄≥65岁的患者中往往未得到充分利用。方法:我们使用公开的CIBMTR P-5640数据集(2008-2019)对慢性期MF - all - hct受体进行了回顾性分析。关键终点包括总生存期(OS)、无病生存期(DFS)、复发、非复发死亡率(NRM)和移植物抗宿主病(GVHD)相关结局。采用Cox回归模型计算风险比(HR)和95%置信区间(CI)。采用R (v4.3.2)进行统计学分析,P < 0.05为差异有统计学意义。结果:我们纳入了856名MF异位hct受体(年龄≥65岁:n = 259,年龄< 65岁:n = 597)。≥65岁患者的2年OS、DFS和无gvhd、无复发生存率(GRFS)分别为62%、33%和12%。复发率、NRM、II-IV级急性GVHD和慢性GVHD分别为43%、27%、46%和42%。在多变量分析中,年龄≥65岁与较差的DFS独立相关(HR 1.26, 95% CI 1.03-1.55, P = 0.025);然而,对移植后的其他结果没有影响。老年患者预后较差的独立预测因素包括脾放疗和ATG使用、继发性MF、较高的合并症(HCT-CI≥3)、供体类型不匹配和骨髓移植。结论:在接受同种异体HCT的65岁及以上骨髓纤维化患者中,与年轻患者相比,DFS较差的2年总生存率为62%,而其他移植后结果保持可比性。这些发现支持allo-HCT作为选择老年MF患者的可行选择,使用量身定制的移植策略。
{"title":"Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Older Patients Aged 65 Years and Above With Myelofibrosis","authors":"Moazzam Shahzad ,&nbsp;Muhammad Kashif Amin ,&nbsp;Sohaib Irfan ,&nbsp;Rania Ahsan ,&nbsp;Hana Qasim ,&nbsp;Muhammad Jawad Javed ,&nbsp;Matthew McGuirk ,&nbsp;Sibgha Gull Chaudhary ,&nbsp;Iqra Anwar ,&nbsp;Abdulraheem Yacoub ,&nbsp;Anurag K. Singh ,&nbsp;Mehdi Hamadani ,&nbsp;Joseph P. Mcguirk ,&nbsp;Muhammad Umair Mushtaq","doi":"10.1016/j.clml.2025.08.012","DOIUrl":"10.1016/j.clml.2025.08.012","url":null,"abstract":"<div><h3>Background</h3><div>Allogeneic hematopoietic stem cell transplantation (allo-HCT) remains the only curative option for myelofibrosis (MF) but is often underutilized in patients aged ≥ 65 due to concerns about treatment-related toxicity.</div></div><div><h3>Methods</h3><div>We conducted a retrospective analysis of chronic-phase MF allo-HCT recipients using the publicly available CIBMTR P-5640 dataset (2008-2019). Key endpoints included overall survival (OS), disease-free survival (DFS), relapse, nonrelapse mortality (NRM), and graft-vs-host disease (GVHD)-related outcomes. Hazard ratios (HR) and 95% confidence intervals (CI) were calculated using Cox regression models. Statistical analysis was performed using R (v4.3.2), with statistical significance defined as <em>P</em> &lt; .05.</div></div><div><h3>Results</h3><div>We included 856 MF allo-HCT recipients (aged ≥ 65: n = 259, aged &lt; 65: n = 597). The 2-year OS, DFS, and GVHD-free, relapse-free survival (GRFS) for patients ≥ 65 years were 62%, 33%, and 12%, respectively. Rates of relapse, NRM, grade II–IV acute GVHD, and chronic GVHD were 43%, 27%, 46%, and 42%, respectively. In multivariable analysis, age ≥ 65 years was independently associated with inferior DFS (HR 1.26, 95% CI 1.03-1.55, <em>P</em> = .025); however, there was no impact on other post-transplant outcomes. Independent predictors of worse outcomes in older patients included splenic radiation and ATG use, secondary MF, higher comorbidities (HCT-CI ≥ 3), mismatched donor type, and bone marrow graft.</div></div><div><h3>Conclusions</h3><div>In myelofibrosis patients aged 65 years and above undergoing allogeneic HCT, a favorable 2-year overall survival of 62% was noted with an inferior DFS compared to younger patients, while other post-transplant outcomes remained comparable. These findings support allo-HCT as a viable option in selected elderly MF patients using tailored transplant strategies.</div></div>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":"26 1","pages":"Pages e32-e40"},"PeriodicalIF":2.7,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145032651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparative Outcomes of Intensive Chemotherapy Versus Venetoclax Plus Hypomethylating Agents in Acute Myeloid Leukemia Patients Aged 60-75 Years: A Propensity Score–Adjusted Cohort Study 60-75岁急性髓系白血病患者强化化疗与Venetoclax加低甲基化药物的比较结果:一项倾向评分调整的队列研究
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.08.016
Moath Albliwi , Emily C. Zabor , John Hanna , Jessica El-Asmar , Daniel P. Nurse , Ameed Bawwab , Hasan Abuamsha , Yomna Abu-Farsakh , Asad Rauf , Heya Batah , Ahmed Mohamed , Muaz Alsabbagh Alchirazi , Akriti G. Jain , John C Molina , Sophia Balderman , Abhay Singh , Aaron T. Gerds , Sudipto Mukherjee , Ronald M. Sobecks , Betty Hamilton , Moaath K. Mustafa Ali

Background

Venetoclax (VEN) combined with hypomethylating agents (HMA) is established as standard of care for elderly or unfit patients with newly diagnosed acute myeloid leukemia (AML). While emerging evidence supports its use in fit, younger patients, data on its comparative efficacy versus intensive chemotherapy (IC) remain limited.

Methods

We conducted a retrospective cohort study to compare composite complete response (CCR), overall survival (OS), and event-free survival (EFS) in patients with AML who received 7 + 3 IC vs. HMA+VEN. All AML patients aged 60–75 years treated between 1/2015 and 9/2023 were included. Propensity score (PS) adjustment and multivariable regression were used to adjust outcomes.

Results

The study included 172 patients who received 7 + 3 and 74 who received HMA+VEN. After adjustment, the average age was –68 years in both groups. The CCR was significantly lower with HMA+VEN (51.4%) than with 7 + 3 (66.9%; P = .025), but this was not significant in the weighted logistic regression model (OR: 0.65, 95% CI: 0.32-1.32, P = .2). PS-adjusted median OS was 18.0 months for 7 + 3 vs. 9.5 months for HMA+VEN (P = .14). VEN+HMA was associated with similar PS-adjusted median EFS (7.2 vs. 7 months, P = .91) compared to 7 + 3. Allogeneic transplant was not different between 7 + 3 and HMA+VEN (48% vs. 24%, log-rank P = .6).

Conclusions

In AML patients aged 60–75 years, HMA + VEN achieved CCR, OS, and EFS comparable to those of the 7 + 3 regimen, consistent with previous reports. However, our analysis may be underpowered or confounded, and a sufficiently powered randomized trial is needed to definitively assess the efficacy, toxicity, and cost-effectiveness of HMA + VEN vs. 7 + 3.
背景:Venetoclax (VEN)联合低甲基化药物(HMA)被确立为老年或新诊断的急性髓性白血病(AML)患者的标准治疗。虽然新出现的证据支持其用于健康的年轻患者,但其与强化化疗(IC)的比较疗效数据仍然有限。方法:我们进行了一项回顾性队列研究,比较接受7 + 3 IC与HMA+VEN治疗的AML患者的综合完全缓解(CCR)、总生存期(OS)和无事件生存期(EFS)。所有在2015年1月至2023年9月期间接受治疗的60-75岁AML患者均被纳入研究。倾向得分(PS)调整和多变量回归对结果进行调整。结果:172例患者接受7 + 3治疗,74例患者接受HMA+VEN治疗。调整后两组患者平均年龄均为-68岁。HMA+VEN组的CCR(51.4%)显著低于7 + 3组(66.9%,P = 0.025),但在加权logistic回归模型中,这一差异无统计学意义(OR: 0.65, 95% CI: 0.32-1.32, P = 0.2)。7 + 3组经ps调整的中位生存期为18.0个月,HMA+VEN组为9.5个月(P = 0.14)。与7 + 3相比,VEN+HMA与ps调整后的中位EFS相似(7.2 vs. 7个月,P = 0.91)。同种异体移植在7 + 3和HMA+VEN之间没有差异(48% vs 24%, log-rank P = .6)。结论:在60-75岁的AML患者中,HMA + VEN获得的CCR、OS和EFS与7 + 3方案相当,与先前的报道一致。然而,我们的分析可能不够有力或存在混淆,需要一项足够有力的随机试验来明确评估HMA + VEN与7 + 3的疗效、毒性和成本效益。
{"title":"Comparative Outcomes of Intensive Chemotherapy Versus Venetoclax Plus Hypomethylating Agents in Acute Myeloid Leukemia Patients Aged 60-75 Years: A Propensity Score–Adjusted Cohort Study","authors":"Moath Albliwi ,&nbsp;Emily C. Zabor ,&nbsp;John Hanna ,&nbsp;Jessica El-Asmar ,&nbsp;Daniel P. Nurse ,&nbsp;Ameed Bawwab ,&nbsp;Hasan Abuamsha ,&nbsp;Yomna Abu-Farsakh ,&nbsp;Asad Rauf ,&nbsp;Heya Batah ,&nbsp;Ahmed Mohamed ,&nbsp;Muaz Alsabbagh Alchirazi ,&nbsp;Akriti G. Jain ,&nbsp;John C Molina ,&nbsp;Sophia Balderman ,&nbsp;Abhay Singh ,&nbsp;Aaron T. Gerds ,&nbsp;Sudipto Mukherjee ,&nbsp;Ronald M. Sobecks ,&nbsp;Betty Hamilton ,&nbsp;Moaath K. Mustafa Ali","doi":"10.1016/j.clml.2025.08.016","DOIUrl":"10.1016/j.clml.2025.08.016","url":null,"abstract":"<div><h3>Background</h3><div>Venetoclax (VEN) combined with hypomethylating agents (HMA) is established as standard of care for elderly or unfit patients with newly diagnosed acute myeloid leukemia (AML). While emerging evidence supports its use in fit, younger patients, data on its comparative efficacy versus intensive chemotherapy (IC) remain limited.</div></div><div><h3>Methods</h3><div>We conducted a retrospective cohort study to compare composite complete response (CCR), overall survival (OS), and event-free survival (EFS) in patients with AML who received 7 + 3 IC vs. HMA+VEN. All AML patients aged 60–75 years treated between 1/2015 and 9/2023 were included. Propensity score (PS) adjustment and multivariable regression were used to adjust outcomes.</div></div><div><h3>Results</h3><div>The study included 172 patients who received 7 + 3 and 74 who received HMA+VEN. After adjustment, the average age was –68 years in both groups. The CCR was significantly lower with HMA+VEN (51.4%) than with 7 + 3 (66.9%; <em>P</em> = .025), but this was not significant in the weighted logistic regression model (OR: 0.65, 95% CI: 0.32-1.32, <em>P =</em> .2). PS-adjusted median OS was 18.0 months for 7 + 3 vs. 9.5 months for HMA+VEN (<em>P =</em> .14). VEN+HMA was associated with similar PS-adjusted median EFS (7.2 vs. 7 months, <em>P =</em> .91) compared to 7 + 3. Allogeneic transplant was not different between 7 + 3 and HMA+VEN (48% vs. 24%, log-rank <em>P =</em> .6).</div></div><div><h3>Conclusions</h3><div>In AML patients aged 60–75 years, HMA + VEN achieved CCR, OS, and EFS comparable to those of the 7 + 3 regimen, consistent with previous reports. However, our analysis may be underpowered or confounded, and a sufficiently powered randomized trial is needed to definitively assess the efficacy, toxicity, and cost-effectiveness of HMA + VEN vs. 7 + 3.</div></div>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":"26 1","pages":"Pages e41-e49"},"PeriodicalIF":2.7,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145063466","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Advanced Multicolor Flow Cytometry Method for Multiple Myeloma 多发性骨髓瘤的先进多色流式细胞术。
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.09.014
Noa Ofir , Ety Rozenberg , Omri Sharabi , Miri Zektser , Ory Rouvio , Roi Gazit

Background

Multiple myeloma (MM) is a bone marrow plasma cells malignancy. Despite impressive advancements of treatments, MM remains incurable due to complex clonality. Here, we present a novel 14-color flow cytometry (FACS) protocol for robust detection and visualization of MM clonality.

Method

We developed a single-tube 14-color FACS protocol to analyze fresh bone marrow samples, tested on 8 MM patients. A premixed, stable antibody cocktail was used to enhance reproducibility and streamline workflow. Dimensionality reduction technique (t-SNE) was applied to visualize plasma cell clonality.

Results

Our panel shown CD38+ CD138+ plasma cells, and their clonality. As expected, MM patients had unique clonal patterns with significant heterogeneity at primary diagnosis. Advanced t-SNE analysis provides a convenient visualization of multi-parameter heterogeneity in a unified 2-D plot. Importantly, our data presents some similarities among patients, and further differences between primary analysis and secondary relapse.

Conclusions

Taken together, our protocol provides a robust and efficient method for improving MM diagnosis using commercially available antibodies and a standard FACS machine. Concise visualization of multi-parameter FACS will help therapeutic decisions and advance the personalized treatment strategies with emerging antigen-specific drugs.
背景:多发性骨髓瘤是一种骨髓浆细胞恶性肿瘤。尽管治疗取得了令人印象深刻的进步,但由于复杂的克隆性,MM仍然无法治愈。在这里,我们提出了一种新的14色流式细胞术(FACS)方案,用于MM克隆的强大检测和可视化。方法:采用单管14色流式细胞仪对8例MM患者的新鲜骨髓样本进行分析。预先混合,稳定的抗体鸡尾酒用于提高重现性和简化工作流程。采用降维技术(t-SNE)可视化浆细胞克隆。结果:我们的小组显示CD38+ CD138+浆细胞及其克隆性。正如预期的那样,MM患者在初次诊断时具有独特的克隆模式和显著的异质性。先进的t-SNE分析在统一的二维图中提供了方便的多参数异质性可视化。重要的是,我们的数据显示了患者之间的一些相似之处,以及原发性分析和继发性复发之间的进一步差异。结论:综上所述,我们的方案提供了一种强大而有效的方法来改善MM的诊断,使用市售抗体和标准的FACS机器。多参数FACS的简明可视化将有助于制定治疗决策,并推进新出现的抗原特异性药物的个性化治疗策略。
{"title":"Advanced Multicolor Flow Cytometry Method for Multiple Myeloma","authors":"Noa Ofir ,&nbsp;Ety Rozenberg ,&nbsp;Omri Sharabi ,&nbsp;Miri Zektser ,&nbsp;Ory Rouvio ,&nbsp;Roi Gazit","doi":"10.1016/j.clml.2025.09.014","DOIUrl":"10.1016/j.clml.2025.09.014","url":null,"abstract":"<div><h3>Background</h3><div>Multiple myeloma (MM) is a bone marrow plasma cells malignancy. Despite impressive advancements of treatments, MM remains incurable due to complex clonality. Here, we present a novel 14-color flow cytometry (FACS) protocol for robust detection and visualization of MM clonality.</div></div><div><h3>Method</h3><div>We developed a single-tube 14-color FACS protocol to analyze fresh bone marrow samples, tested on 8 MM patients. A premixed, stable antibody cocktail was used to enhance reproducibility and streamline workflow. Dimensionality reduction technique (t-SNE) was applied to visualize plasma cell clonality.</div></div><div><h3>Results</h3><div>Our panel shown CD38<sup>+</sup> CD138<sup>+</sup> plasma cells, and their clonality. As expected, MM patients had unique clonal patterns with significant heterogeneity at primary diagnosis. Advanced t-SNE analysis provides a convenient visualization of multi-parameter heterogeneity in a unified 2-D plot. Importantly, our data presents some similarities among patients, and further differences between primary analysis and secondary relapse.</div></div><div><h3>Conclusions</h3><div>Taken together, our protocol provides a robust and efficient method for improving MM diagnosis using commercially available antibodies and a standard FACS machine. Concise visualization of multi-parameter FACS will help therapeutic decisions and advance the personalized treatment strategies with emerging antigen-specific drugs.</div></div>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":"26 1","pages":"Pages e132-e140.e5"},"PeriodicalIF":2.7,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145426722","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Bispecific Antibody Therapy for Multiple Myeloma: A Practical Toolkit. 双特异性抗体治疗多发性骨髓瘤:一个实用的工具包。
IF 2.7 4区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-26 DOI: 10.1016/j.clml.2025.12.015
Cesar Rodriguez, Cyrille Touzeau, Edvan de Queiroz Crusoe, Fernando Vieira Pericole, Vania Hungria

Purpose: Bispecific antibodies emerged as a new class of treatment for patients with relapsed/refractory multiple myeloma. Education on the practicalities of delivery and patient management is vital to optimize outcomes with these novel agents.

Methods: A group of 5 experienced hematologists from centers in the United States, Europe and Latin America met to discuss key considerations for bispecific antibody therapy and agree on best practice advice.

Results and discussion: This paper presents advice for best practice at each stage of the bispecific antibody therapy patient journey: patient eligibility, pretreatment medication, step-up dosing, treatment, management of adverse events and long-term care.

Conclusion: We believe that the expert insights presented here will help to educate healthcare professionals and optimize patient outcomes by guiding the implementation of bispecific antibody therapy into real-world practice.

目的:双特异性抗体作为一种新的治疗复发/难治性多发性骨髓瘤的方法出现。教育的实用性交付和患者管理是至关重要的,以优化这些新型药物的结果。方法:一组来自美国、欧洲和拉丁美洲中心的5名经验丰富的血液学家开会讨论双特异性抗体治疗的关键因素,并就最佳实践建议达成一致。结果和讨论:本文提出了双特异性抗体治疗患者旅程的每个阶段的最佳实践建议:患者资格,预处理药物,增加剂量,治疗,不良事件管理和长期护理。结论:我们相信,这里提出的专家见解将有助于教育医疗保健专业人员,并通过指导双特异性抗体治疗在现实世界中的实施来优化患者的结果。
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引用次数: 0
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Clinical Lymphoma, Myeloma & Leukemia
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