Pediatric Blood & Cancer最新文献

{"title":"The benefits of prophylactic defibrotide: Are the tides turning?","authors":"Mahvish Q. Rahim,&nbsp;April L. Rahrig,&nbsp;Devin Dinora,&nbsp;Jessica Harrison,&nbsp;Ryanne Green,&nbsp;Allie Carter,&nbsp;Jodi Skiles","doi":"10.1002/pbc.31396","DOIUrl":"10.1002/pbc.31396","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Veno-occlusive disease (VOD) is a life-threatening endotheliopathy that can occur after stem cell transplant (SCT). Numerous risk factors contribute to the development of VOD during SCT, and the role of prophylactic defibrotide (DF) in mitigating these risks remains unclear.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>We compare not only the incidence of VOD development, but also the severity of VOD and survival outcomes between patients who did and did not develop VOD and did or did not receive prophylactic DF.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Study design</h3>\u0000 \u0000 <p>In this single-center retrospective study of 58 pediatric SCT patients from 2008 to 2022, we compare the demographics, risk profiles, and outcomes within three cohorts: Group 1: prophylactic DF and no VOD (<i>n</i> = 5), Group 2: prophylactic DF and development of VOD (<i>n</i> = 6), and Group 3: treatment DF for patients who developed VOD (<i>n</i> = 47).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Patients with VOD who did not receive prophylactic DF had higher severity classification of disease at onset (very severe 80.9% vs. 66.7%, <i>p</i> = .592) and at maximum severity (very severe 89.4% vs. 83.3%, <i>p</i> = .532), as opposed to mild, moderate, or severe categorization compared to those who did not receive prophylactic DF. Patients who developed VOD and did not receive prophylactic DF had a lower 1-year survival probability compared to those who received prophylactic DF and still developed VOD (51.1% vs. 75% alive at 1 year, excluding the two subjects without adequate follow-up time, <i>p</i> = .266).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Although, not statistically significant in our small retrospective study, there is potential overall survival and decreased VOD severity benefits of prophylactic DF.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31396","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142564494","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Use of plerixafor for short-term management of leukopenia in a pediatric patient with WHIM syndrome","authors":"Beatriz Caceres-Nazario,&nbsp;Kimberly A. Kasow,&nbsp;Staci Keene,&nbsp;Joshua J. Bies,&nbsp;Maria Boucher","doi":"10.1002/pbc.31401","DOIUrl":"10.1002/pbc.31401","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142546722","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Gastrointestinal Stromal Tumor With Extensive Gastric Polymorphic Polyposis: A Potential Relationship?","authors":"Prasiksha Sitaula,&nbsp;Janet M. Poulik,&nbsp;Mohammad F. El-Baba,&nbsp;Justin Klein,&nbsp;Manisha Gadgeel,&nbsp;John W. Glod,&nbsp;Severyn Ferneza,&nbsp;Süreyya Savaşan","doi":"10.1002/pbc.31416","DOIUrl":"10.1002/pbc.31416","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142546721","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A soaring price, a silent fight: Thioguanine portends new access barriers to life-saving treatments for children with cancer","authors":"David S. Dickens,&nbsp;Jason Cleppe","doi":"10.1002/pbc.31410","DOIUrl":"10.1002/pbc.31410","url":null,"abstract":"&lt;p&gt;Global access to cancer medicines is a well-documented barrier to care in low- and middle-income countries.&lt;span&gt;&lt;sup&gt;1, 2&lt;/sup&gt;&lt;/span&gt; Availability, affordability, access, and pricing repeatedly arise as barriers internationally with wide variability between regions. In the United States, a 2021 cross-sectional study revealed disparities in access to pediatric cancer care along racial, ethnic, geographic, and socioeconomic groups.&lt;span&gt;&lt;sup&gt;3&lt;/sup&gt;&lt;/span&gt; Additionally, chemotherapy drug shortages have continued to present a significant challenge to access for all,&lt;span&gt;&lt;sup&gt;4&lt;/sup&gt;&lt;/span&gt; particularly for sterile injectable, generic products. In the year 2024, pediatric oncologists have been faced with a new barrier to access for an essential component of leukemia therapy.&lt;/p&gt;&lt;p&gt;Thioguanine was approved by the United States Food and Drug Administration in 1966 for remission induction and remission consolidation treatment of acute nonlymphocytic leukemias and has not changed since.&lt;span&gt;&lt;sup&gt;5&lt;/sup&gt;&lt;/span&gt; In 1993, the Children's Cancer Group confirmed in a pivotal Phase III trial that the addition of a phase of care known as “Delayed Intensification (DI)” (inclusive of 2 weeks of thioguanine) significantly improved outcomes in acute lymphoblastic leukemia.&lt;span&gt;&lt;sup&gt;6&lt;/sup&gt;&lt;/span&gt; Numerous large, cooperative group trials have since been published, which validate the importance of DI as a component of standard of care therapy for pediatric patients with acute lymphoblastic leukemia, which continues today.&lt;/p&gt;&lt;p&gt;Recently, thioguanine manufacturing was transitioned from West-Ward Pharmaceuticals PLC (now the Hikma Group) to Waylis Therapeutics LLC. In that transition, two things happened. One, the price per tablet (Tabloid®) increased by 20-fold. What used to cost an average of ∼800 US dollars per 2-week course is now averaging ∼17,000 USD. Second, Waylis has yet to enter Tabloid® into the Medicaid Prescription Drug Rebate Program. Without this step, state Medicaid officials cannot recognize Tabloid® as a covered entity. At the previous cost, exceptions to policy were routinely granted in the state of Iowa. However with current pricing, this is no longer possible. Instead, families are directed to Waylis’ patient access program, designed for patients without insurance or live at or below 500% of the current federal poverty level. Despite the burden and time of the paperwork, with adequate advanced planning, we have been able to secure thioguanine for all our patients. Unfortunately, some sites have informally reported thioguanine omission or substitution with mercaptopurine. Though this substitution is sensible, previous data from the Children's Oncology Group Phase III study CCG-1952 showed better EFS when thioguanine was used in maintenance when compared with mercaptopurine.&lt;span&gt;&lt;sup&gt;7&lt;/sup&gt;&lt;/span&gt; Though this approach was abandoned due to toxicity, the study is added evidence that these two related medicines are not entirely equivalent. As t","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31410","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142505512","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Recording diagnostic conversations for communication research purposes in pediatric leukemia","authors":"Petra Buursma,&nbsp;Sasja A. Schepers,&nbsp;Marijke C. Kars,&nbsp;Esther M. M. van den Bergh,&nbsp;Natasja Dors,&nbsp;Martha A. Grootenhuis,&nbsp;Peter M. Hoogerbrugge","doi":"10.1002/pbc.31395","DOIUrl":"10.1002/pbc.31395","url":null,"abstract":"<p>Recordings of patient–doctor interactions is a recommended method in communication research. However, concerns are expressed regarding audio-recording of conversations with vulnerable patients. Our study examined experiences of children, parents, and oncologists with recording diagnostic conversations in the pediatric acute leukemia setting. Results show that recording conversations is generally well received by virtually all children and parents. Pediatric oncologists seem to overestimate the expected emotional burden for children and parents, which may lead to gatekeeping by professionals. This in turn may lead to a decrease in patient autonomy and research quality when addressing relevant questions in communication science.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31395","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142505514","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Case report: Dichloroacetate-induced methaemoglobinaemia in a G6PD-deficient neonate","authors":"Ze Lei Tan,&nbsp;Nicholas Beng Hui Ng,&nbsp;Jacqueline Soo May Ong","doi":"10.1002/pbc.31408","DOIUrl":"10.1002/pbc.31408","url":null,"abstract":"<p>A 3-week-old neonate with glucose-6-phosphate dehydrogenase (G6PD) deficiency and primary lactic acidosis developed haemolytic jaundice and methaemoglobinaemia following treatment with dichloroacetate (DCA), a standard treatment for primary lactic acidosis. While this mechanism has been reported in the sheep model, it has not been described in humans. Our case reinforces the uncommon observation that a G6PD-deficient individual experiencing oxidative stress may develop concurrent methaemoglobinaemia. In this case, methylene blue, the standard treatment for methaemoglobinaemia, may result in further oxidative stress. The judicious use of blood transfusion to correct the oxygen-carrying capacity of our patient led to reversal of the methaemoglobinaemia.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142505513","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcome of childhood ALK-positive anaplastic large cell lymphoma relapses: Real-life experience of the French Society of Pediatric Oncology (SFCE) cohort of 75 French children","authors":"Victor Pereira,&nbsp;Maël Barthoulot,&nbsp;Nathalie Aladjidi,&nbsp;Audrey Contet,&nbsp;Jean-Hugues Dalle,&nbsp;Marie Émilie Dourthe,&nbsp;Nathalie Garnier,&nbsp;Bénédicte Bruno,&nbsp;Amaury Leruste,&nbsp;Isabelle Pellier,&nbsp;Matthieu Simonin,&nbsp;Catherine Paillard,&nbsp;Arnauld Verschuur,&nbsp;Stéphane Ducassou,&nbsp;Laurence Lamant,&nbsp;Laurence Brugieres,&nbsp;Marie-Cécile Le Deley,&nbsp;Charlotte Rigaud","doi":"10.1002/pbc.31397","DOIUrl":"10.1002/pbc.31397","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>To describe treatments and outcomes of French children treated for relapsed/refractory anaplastic lymphoma kinase-positive anaplastic large cell lymphoma (ALK+ ALCL).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted the analysis of a series of 75 French children treated for a first relapsed/refractory ALK+ ALCL between 1999 and 2017.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The median time to first relapse was 8.1 months from initial diagnosis (2.9 after end of treatment), with 12 relapses during frontline treatment or within 1 month of the end of treatment. Treatment of the first relapse varied according to the period of time and risk factors: 48 received multiagent chemotherapy, including 21 and 19 consolidated with allogeneic stem cell transplantation (SCT) and autologous-SCT, respectively. Twenty-one patients received weekly vinblastine, and six received ALK inhibitors (ALKi). Overall, 64/75 patients reached a second complete remission (CR2). Eight out of 11 patients who did not reach CR2 died and the other three were rescued with ALKi, vinblastine, and nivolumab. With a median follow-up of 8.2 years, 60 patients are alive, 43 in CR2, 15 in CR3, two in CR4; and 15 patients died, six from toxicity and nine from disease progression. The 5-year event-free survival and overall survival after first relapse were 51.7% (95% confidence interval [CI]: 39.6%–62.6%) and 80.7% (95% CI: 69.6%–88.1%), respectively. Time to relapse greater than 12 months from initial diagnosis was proven to be a prognostic factor in relapsed/refractory ALK+ ALCL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>In relapsed ALK+ ALCL, high survival rate can be reached with various therapeutic strategies. The main challenge remains to prevent subsequent relapses, and to lower long-term morbidity.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31397","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472051","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Relapse and survival after relapse among children with cancer in Denmark: 2001–2021","authors":"Mie Mølgaard Andersen,&nbsp;Marie Christine Lundius Sørensen,&nbsp;Kjeld Schmiegelow,&nbsp;Astrid Marie Sehested,&nbsp;Klaus Rostgaard,&nbsp;Marianne Olsen,&nbsp;Torben Stamm Mikkelsen,&nbsp;Peder Skov Wehner,&nbsp;Lisa Lyngsie Hjalgrim,&nbsp;Signe Holst Søegaard","doi":"10.1002/pbc.31384","DOIUrl":"10.1002/pbc.31384","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>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.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>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.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>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 (<i>p</i> = .07), and from 21% to 18% among children with solid tumours (<i>p</i> = .26). Concurrently, the 5-year survival after relapse increased among children with haematological cancers (from 44% to 61%, <i>p</i> = .03) and solid tumours (from 38% to 46%, <i>p</i> = .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%, <i>p</i> = .82; and 20% and 18%, <i>p</i> = .90) comparing 2010–2015 and 2016–2021.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>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.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31384","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472057","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association of inpatient and outpatient pediatric palliative care with healthcare utilization and end-of-life outcomes in pediatric oncology","authors":"Rebecca L. Shamah,&nbsp;Elizabeth George,&nbsp;Nicholas P. DeGroote,&nbsp;Karen Wasilewski,&nbsp;Katharine E. Brock","doi":"10.1002/pbc.31387","DOIUrl":"10.1002/pbc.31387","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>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.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>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.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>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 (<i>p</i> &lt; .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 <i>p</i> &lt; .001), and had more intensive medical interventions performed (<i>p</i> &lt; .01). Outpatient PPC recipients were less likely to receive intravenous (IV) chemotherapy (<i>p</i> &lt; .01) or intubation (<i>p</i> = .05), and more likely to receive hospice, die at home, and have an outpatient do-not-resuscitate order (all <i>p</i> &lt; .001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>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.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472028","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost–utility of nelarabine for the first-line treatment of newly diagnosed pediatric T-cell acute lymphoblastic leukemia in Canada","authors":"Roaa Shoukry,&nbsp;Alexandra Moskalewicz,&nbsp;Nicole Bradley,&nbsp;Elizabeth Bond,&nbsp;Mandy Sala,&nbsp;Sumit Gupta,&nbsp;Paul Gibson,&nbsp;Petros Pechlivanoglou","doi":"10.1002/pbc.31393","DOIUrl":"10.1002/pbc.31393","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>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.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>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.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>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.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Incorporating nelarabine into aBFM was cost-effective across different scenarios and assumptions. These results support its funding by public and private payers.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31393","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472041","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}