Pediatric Blood & Cancer最新文献

英文中文

Relapse and survival after relapse among children with cancer in Denmark: 2001–2021 丹麦癌症儿童的复发率和复发后的存活率：2001-2021 年。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-21 DOI: 10.1002/pbc.31384

Mie Mølgaard Andersen, Marie Christine Lundius Sørensen, Kjeld Schmiegelow, Astrid Marie Sehested, Klaus Rostgaard, Marianne Olsen, Torben Stamm Mikkelsen, Peder Skov Wehner, Lisa Lyngsie Hjalgrim, Signe Holst Søegaard

Background

In recent decades, new first and subsequent lines of anticancer treatment and supportive care have improved survival for children with cancer. We investigated recent temporal changes in the incidence of relapse and survival after relapse among children with cancer in Denmark.

Procedure

This register-based study included 2890 children diagnosed before age 15 years with haematological cancers and solid tumours (2001–2021) and central nervous system (CNS) tumours (2010–2021). We used the Aalen–Johansen and Kaplan–Meier estimators to assess cumulative incidence of relapse—defined as cancer recurrence or progression—and survival probability after relapse.

Results

Comparing the periods 2001–2010 and 2011–2021, the 5-year cumulative incidence of relapse decreased from 14% to 11% among children with haematological cancers (p = .07), and from 21% to 18% among children with solid tumours (p = .26). Concurrently, the 5-year survival after relapse increased among children with haematological cancers (from 44% to 61%, p = .03) and solid tumours (from 38% to 46%, p = .25). Among children with malignant CNS tumours, the 5-year cumulative incidence of relapse and the 5-year survival after relapse remained stable (49% and 51%, p = .82; and 20% and 18%, p = .90) comparing 2010–2015 and 2016–2021.

Conclusions

In recent decades in Denmark, improvements were observed in reducing relapse incidence and increasing survival after relapse in children with haematological cancers and solid tumours. However, the persistent survival gap between children who relapse and those who do not across all childhood cancers underlines the need for intensified and highly targeted treatments for children at high risk of relapse.

背景：近几十年来，新的一线和后续抗癌治疗以及支持性护理改善了癌症儿童的生存状况。我们调查了丹麦癌症儿童复发率和复发后存活率的近期时间变化：这项以登记为基础的研究纳入了 2890 名 15 岁前确诊患有血液肿瘤和实体瘤（2001-2021 年）以及中枢神经系统（CNS）肿瘤（2010-2021 年）的儿童。我们采用Aalen-Johansen和Kaplan-Meier估计法评估复发（即癌症复发或进展）的累积发生率以及复发后的生存概率：结果：与2001-2010年和2011-2021年相比，血液肿瘤儿童的5年累积复发率从14%降至11%（p = .07），实体瘤儿童的5年累积复发率从21%降至18%（p = .26）。同时，血液肿瘤和实体瘤患儿的复发后5年生存率也有所提高，前者从44%提高到61%（p = .03），后者从38%提高到46%（p = .25）。在中枢神经系统恶性肿瘤患儿中，与2010-2015年和2016-2021年相比，复发的5年累积发生率和复发后的5年生存率保持稳定（49%和51%，p = .82；20%和18%，p = .90）：近几十年来，丹麦在降低血液肿瘤和实体瘤患儿的复发率和提高复发后存活率方面取得了进步。然而，在所有儿童癌症中，复发儿童与未复发儿童之间的生存率差距依然存在，这凸显了对高复发风险儿童加强高度针对性治疗的必要性。

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引用次数: 0

Association of inpatient and outpatient pediatric palliative care with healthcare utilization and end-of-life outcomes in pediatric oncology 住院和门诊儿科姑息关怀与儿科肿瘤的医疗利用率和临终结局的关系。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-20 DOI: 10.1002/pbc.31387

Rebecca L. Shamah, Elizabeth George, Nicholas P. DeGroote, Karen Wasilewski, Katharine E. Brock

Background

Pediatric palliative care (PPC) is associated with improved end-of-life (EOL) outcomes. Inpatient and outpatient PPC have unique roles during the disease course. Yet, it is unknown whether the location of PPC receipt (inpatient vs. outpatient) is associated with healthcare utilization and EOL outcomes for pediatric and adolescent and young adult oncology patients.

Procedure

A retrospective single-institution chart review of pediatric patients (age 0–28) with cancer who died between January 2015 and December 2022 was performed to compare EOL outcomes and healthcare utilization metrics among inpatient PPC, any outpatient PPC, and non-PPC recipients. Demographics and clinical factors were analyzed by PPC receipt location.

Results

Among 450 patients, 292 (64.9%) received PPC (inpatient only 35%, any outpatient 65%). Patients who died without receiving PPC dropped from 69% to 22% following development of an outpatient PPC clinic (p < .001). In the last 6 months, 1 month, and last week of life, inpatient PPC recipients spent more days admitted to the hospital and intensive care unit (all p < .001), and had more intensive medical interventions performed (p < .01). Outpatient PPC recipients were less likely to receive intravenous (IV) chemotherapy (p < .01) or intubation (p = .05), and more likely to receive hospice, die at home, and have an outpatient do-not-resuscitate order (all p < .001).

Conclusions

PPC receipt substantially increased after the creation of an outpatient PPC clinic, suggesting that outpatient PPC is critical in the provision of PPC to children with cancer. Outpatient PPC was associated with fewer hospital days, IV chemotherapy, and intubation at EOL, while increasing hospice enrollment and home death.

背景：儿科姑息治疗（PPC）与改善生命末期（EOL）预后有关。住院病人和门诊病人的姑息治疗在疾病过程中发挥着独特的作用。然而，接受姑息治疗的地点（住院病人与门诊病人）是否与儿科、青少年和年轻成人肿瘤患者的医疗利用率和临终结局有关，目前尚不得而知：对 2015 年 1 月至 2022 年 12 月期间死亡的儿科癌症患者（0-28 岁）进行了单机构病历回顾，以比较住院患者、任何门诊患者和非住院患者的临终结局和医疗利用指标。人口统计学和临床因素按PPC接受地点进行了分析：在 450 名患者中，292 人（64.9%）接受了 PPC 治疗（住院患者占 35%，任何门诊患者占 65%）。在设立 PPC 门诊后，未接受 PPC 治疗而死亡的患者比例从 69% 降至 22%（p 结论：PPC 门诊的设立大大提高了患者接受 PPC 治疗的比例：门诊患者 PPC 诊所成立后，接受 PPC 治疗的患者大幅增加，这表明门诊患者 PPC 对为癌症儿童提供 PPC 治疗至关重要。门诊就诊与减少住院天数、静脉化疗和临终前插管有关，同时也增加了临终关怀的注册人数和居家死亡人数。

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引用次数: 0

Cost–utility of nelarabine for the first-line treatment of newly diagnosed pediatric T-cell acute lymphoblastic leukemia in Canada 在加拿大，奈拉滨用于新诊断的小儿 T 细胞急性淋巴细胞白血病一线治疗的成本效益。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-20 DOI: 10.1002/pbc.31393

Roaa Shoukry, Alexandra Moskalewicz, Nicole Bradley, Elizabeth Bond, Mandy Sala, Sumit Gupta, Paul Gibson, Petros Pechlivanoglou

Background

The Children's Oncology Group (COG)-AALL0434 trial investigated the addition of nelarabine to the augmented Berlin–Frankfurt–Münster (aBFM) protocol in patients (1.0–30.99 years) with newly diagnosed T-cell acute lymphoblastic leukemia (T-ALL). Despite demonstrating superior outcomes, nelarabine is not currently funded by many health systems, in part due to a lack of cost-effectiveness data. We estimated the cost–utility of nelarabine for this indication from a Canadian public healthcare payer perspective.

Methods

We developed a microsimulation model that followed hypothetical patients with newly diagnosed T-ALL from post-induction therapy to death. Three health states were modeled: relapse-free, post-relapse, and death. Efficacy was estimated using AALL0434 and retrospective data from Ontario, Canada. Costs were obtained from Canadian sources. Utility estimates and long-term mortality risks were sourced from literature. Total healthcare costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER) were reported. Probabilistic and scenario analyses were conducted.

Results

Incorporating nelarabine in the aBFM protocol increased costs by $51,670 Canadian dollars per patient, but resulted in 1.97 more QALYs and an ICER of $26,184/QALY. Most of the identified cost and benefit were accrued within the AALL0434 trial period (first 11 years post diagnosis) and while patients were in the relapse-free health state. Across multiple scenarios, the ICER was stable under an assumed $50,000/QALY threshold.

Conclusion

Incorporating nelarabine into aBFM was cost-effective across different scenarios and assumptions. These results support its funding by public and private payers.

背景：儿童肿瘤组织（COG）-AALL0434试验研究了在新诊断的T细胞急性淋巴细胞白血病（T-ALL）患者（1.0-30.99岁）的柏林-法兰克福-明斯特（aBFM）增强方案中加入奈拉滨的情况。尽管尼拉拉滨的疗效显著，但目前许多医疗系统并不资助它，部分原因是缺乏成本效益数据。我们从加拿大公共医疗支付方的角度估算了用于该适应症的奈拉拉滨的成本效用：我们建立了一个微观模拟模型，该模型跟踪假定的新诊断 T-ALL 患者从诱导治疗后到死亡的整个过程。模型模拟了三种健康状态：无复发、复发后和死亡。使用 AALL0434 和加拿大安大略省的回顾性数据估算疗效。成本来自加拿大。效用估计值和长期死亡风险来自文献。报告了总医疗成本、质量调整生命年 (QALY) 和增量成本效益比 (ICER)。进行了概率和情景分析：在 aBFM 方案中加入奈拉滨会使每名患者的成本增加 51,670 加元，但 QALYs 增加了 1.97，ICER 为 26,184 加元/QALY。大部分已确定的成本和收益都是在 AALL0434 试验期间（诊断后的前 11 年）以及患者处于无复发健康状态时累积的。在多种方案中，ICER 在假定的 50,000 美元/QALY 临界值下保持稳定：结论：在不同的方案和假设下，将奈拉拉滨纳入生物燃料管理具有成本效益。这些结果支持公共和私人付费者对其进行资助。

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引用次数: 0

Feasibility of transcranial Doppler to evaluate vasculopathy among survivors of childhood brain tumors exposed to cranial radiation therapy 经颅多普勒评估接受头颅放射治疗的儿童脑肿瘤幸存者血管病变的可行性。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-20 DOI: 10.1002/pbc.31392

Daniel C. Bowers, Mark D. Johnson

Background

The ability of transcranial Doppler (TCD) to detect asymptomatic cerebrovascular disease among childhood brain tumor survivors following exposure to cranial radiation therapy has not been established.

Methods

Survivors of childhood brain tumors, more than 3 years since diagnosis and exposed to greater than 30 Gy cranial radiation, underwent a history and physical exam, laboratory biomarkers of cerebrovascular disease (cholesterol, high-density lipoprotein cholesterol (HDL), low-density lipoprotein cholesterol (LDL), high-sensitivity CRP, hemoglobin A1C, apoprotein A, and apoprotein B), and a TCD evaluation of their cerebral arteries.

Results

In all 165 cerebral arteries from 13 patients (medulloblastoma = 10; germ cell tumor = 3; females = 5; mean age at diagnosis = 8.0 years; mean age at time of study = 20.9 years) were examined. Twenty-eight of 165 (17%) were considered abnormal by pre-specified criteria. Total 114 cerebral arteries from 13 patients were assessed for greater than 50% stenosis velocities. Arteries most likely to be considered abnormal included the distal bilateral vertebral arteries (right 38%, left 30%), basilar artery 30%, bilateral siphon internal carotid arteries (right 30%, left 23%), bilateral middle cerebral arteries (23% bilaterally), and bilateral anterior cerebral arteries (7% bilaterally). Two vessels had mean flow velocities consistent with $� � \geq � �$ 50% stenosis (1.8%). No vessels were found to have greater than 80% stenosis.

Conclusions

TCD may be a useful and practical tool to examine asymptomatic cerebrovascular disease among childhood brain tumor survivors after exposure to cranial radiation therapy. Posterior circulation vessels appear to have the highest burden of disease in this group of brain tumor survivors, a majority of whom had medulloblastoma.

背景：经颅多普勒（TCD）检测儿童脑肿瘤幸存者接受颅内放射治疗后无症状脑血管疾病的能力尚未确定：经颅多普勒（TCD）检测接受头颅放射治疗的儿童脑肿瘤幸存者无症状脑血管疾病的能力尚未得到证实：方法：诊断后超过 3 年且接受过超过 30 Gy 头颅放射治疗的儿童脑肿瘤幸存者接受病史和体格检查、脑血管疾病的实验室生物标志物（胆固醇、高密度脂蛋白胆固醇 (HDL)、低密度脂蛋白胆固醇 (LDL)、高敏 CRP、血红蛋白 A1C、载脂蛋白 A 和载脂蛋白 B）以及脑动脉的 TCD 评估：共检查了 13 名患者（髓母细胞瘤 = 10；生殖细胞瘤 = 3；女性 = 5；诊断时平均年龄 = 8.0 岁；研究时平均年龄 = 20.9 岁）的 165 条脑动脉。根据预先指定的标准，165 例中有 28 例（17%）被认为是异常的。共有 13 名患者的 114 条脑动脉的狭窄速度超过 50%。最有可能被认为异常的动脉包括双侧椎动脉远端（右侧 38%，左侧 30%）、基底动脉 30%、双侧虹吸型颈内动脉（右侧 30%，左侧 23%）、双侧大脑中动脉（双侧 23%）和双侧大脑前动脉（双侧 7%）。两根血管的平均流速符合≥ $ge $50%的狭窄程度（1.8%）。没有发现血管狭窄超过80%：TCD可能是检查儿童脑肿瘤幸存者在接受头颅放射治疗后无症状脑血管疾病的一种有用而实用的工具。在这组脑肿瘤幸存者中，后循环血管的疾病负担似乎最重，其中大多数人患有髓母细胞瘤。

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引用次数: 0

How you play the game 如何玩游戏

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-18 DOI: 10.1002/pbc.31405

Andrea Watson

引用次数: 0

Reply to: Comment on: Perinatal risk factors and neurocognitive outcomes in children and adolescents with sickle cell disease 答复评论：患有镰状细胞病的儿童和青少年的围产期风险因素和神经认知结果。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-18 DOI: 10.1002/pbc.31391

Jennifer Longoria, Andrew Heitzer, Jane Hankins

引用次数: 0

Comment on: Perinatal risk factors and neurocognitive outcomes in children and adolescents with sickle cell disease 评论：患有镰状细胞病的儿童和青少年的围产期风险因素和神经认知结果。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-18 DOI: 10.1002/pbc.31318

Wei-Zhen Tang, Wen-Xin Deng, Tai-Hang Liu

引用次数: 0

Correction to “Delivery of re-irradiation and complex palliative radiotherapy using proton therapy in pediatric cancer patients” 对 "利用质子疗法为儿科癌症患者提供再照射和复合姑息放疗 "的更正。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-18 DOI: 10.1002/pbc.31403

Berlin E, Eisenberg R, Hill-Kayser C, et al. Delivery of re-irradiation and complex palliative radiotherapy using proton therapy in pediatric cancer patients. Pediatr Blood Cancer. 2023;70:e30708. doi:10.1002/pbc.30708

Eva Berlin and Rachel Eisenberg should be listed as co-first authors and equal contribution.

The author byline should read:

This should have read “Eva Berlin*, Rachel Eisenberg*, Christine Hill-Kayser, Robert A. Lustig, Goldie Kurtz, Elizabeth Cummings, Michael LaRiviere *Co-first authors”

We apologize for this error.

Berlin E、Eisenberg R、Hill-Kayser C 等：利用质子疗法对儿科癌症患者进行再照射和复合姑息放疗。Pediatr Blood Cancer.2023;70:e30708。doi:10.1002/pbc.30708Eva Berlin和Rachel Eisenberg应列为共同第一作者，且贡献相同。作者署名应为：应为 "Eva Berlin*、Rachel Eisenberg*、Christine Hill-Kayser、Robert A. Lustig、Goldie Kurtz、Elizabeth Cummings、Michael LaRiviere *共同第一作者"，我们对此错误深表歉意。

引用次数: 0

Upfront immunotherapy for synchronous high-grade glioma and B-lymphoma in a pediatric patient with CMMRD syndrome 对一名患有CMMRD综合征的儿科患者进行同步高级别胶质瘤和B淋巴瘤的前期免疫治疗。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-18 DOI: 10.1002/pbc.31399

Thérèse De Vanssay, Nour El Riachy, Caroline Donze, Romain Appay, Didier Scavarda, Benoit Testud, Nicolas Andre, Gabriel Revon-Rivière

引用次数: 0

Prevent Acute Chest Syndrome checklist (PACScheck): A quality improvement initiative to reduce acute chest syndrome 预防急性胸部综合征检查表（PACScheck）：减少急性胸部综合征的质量改进措施。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2024-10-17 DOI: 10.1002/pbc.31378

Kerry Morrone, Kaitlin Strumph, Catherine Pisacano, Jessica Briggs, Rachelle Zipper, Bhaumik B. Patel, Susanna Chang, Wen-Ling Kyon, Kristen Ronca, Miranda Abyazi, Geoffrey Cheng, Leya Schwartz, Jennifer De Los Santos, Janine Keenan, Marina Reznik, Deepa Manwani, Michael L. Rinke

Background

Acute chest syndrome (ACS) is a life-threatening complication of sickle cell disease (SCD). The Prevent Acute Chest Syndrome checklist (PACScheck) was created to drive appropriate ordering of opioids, incentive spirometry (IS), intravenous fluids (IVF), evaluation of oxygen desaturation, and bronchodilator use.

Objectives

Decrease the development of ACS by 5% in a hospitalized pediatric SCD population.

Methods

A multidisciplinary team conducted a quality improvement (QI) project between April 2020 and August 2021 on an inpatient pediatric hematology unit. At-risk hospitalizations were patients with SCD who did not have ACS upon hospital admission. PACScheck was implemented and weekly run charts assessed documentation. Process control (p) charts, geometric control (g) charts, and chi-square tests assessed checklist process measures pre- and post-PACScheck. G chart assessed the number of encounters between ACS events.

Results

A total of 483 at-risk hospitalizations were identified in the 12 months prior and 363 during the study period. A g chart demonstrated that fewer encounters developed ACS during PACScheck. A p chart demonstrated that IS documentation increased during PACScheck. A run chart of PACScheck documentation demonstrated a median of 100% documentation at least once per hospitalization during the last six months of the intervention.

Conclusion

Development of ACS can be reduced by implementing a best-practices checklist (PACScheck) on an inpatient pediatric hematology unit with a multidisciplinary team.

背景：急性胸部综合征 (ACS) 是镰状细胞病 (SCD) 的一种危及生命的并发症。创建预防急性胸部综合征检查表（PACScheck）的目的是推动阿片类药物、激励肺活量测定（IS）、静脉输液（IVF）、氧饱和度评估和支气管扩张剂使用的合理下单：目标：将儿科 SCD 住院患者的 ACS 发生率降低 5%：2020年4月至2021年8月期间，一个多学科团队在儿科血液科住院病房开展了一项质量改进（QI）项目。高危住院患者是指入院时未出现 ACS 的 SCD 患者。实施了 PACScheck，并每周对运行图进行评估记录。流程控制（P）图、几何控制（G）图和卡方检验评估了 PACScheck 前后的检查单流程措施。G 图表评估了两次 ACS 事件之间的接触次数：结果：研究前 12 个月共发现 483 例高风险住院病例，研究期间发现 363 例。G 图表显示，在 PACScheck 期间发生 ACS 的就诊次数减少。P图显示，PACScheck期间IS记录有所增加。PACScheck 记录的运行图显示，在干预的最后六个月中，每次住院至少一次记录的中位数为 100%：结论：在拥有多学科团队的儿科血液科住院病房实施最佳实践检查表（PACScheck）可减少 ACS 的发生。

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