Pediatric Transplantation最新文献

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Chronotropic Response to Exercise is Decreased in Patients With Congenital Heart Disease Compared to Cardiomyopathy Following Pediatric Heart Transplantation 与心肌病患者相比,先天性心脏病患者在接受小儿心脏移植手术后对运动的顺时针反应减弱
IF 1.3 4区 医学
Pediatric Transplantation Pub Date : 2024-09-13 DOI: 10.1111/petr.14856
Sebastian Otto‐Meyer, Alan P. Wang, Garett J. Griffith, Katheryn Gambetta, Kendra Ward
{"title":"Chronotropic Response to Exercise is Decreased in Patients With Congenital Heart Disease Compared to Cardiomyopathy Following Pediatric Heart Transplantation","authors":"Sebastian Otto‐Meyer, Alan P. Wang, Garett J. Griffith, Katheryn Gambetta, Kendra Ward","doi":"10.1111/petr.14856","DOIUrl":"https://doi.org/10.1111/petr.14856","url":null,"abstract":"BackgroundTwo common indications for pediatric heart transplantation are congenital heart disease and cardiomyopathy. Prior studies suggest differences in chronotropy on cardiopulmonary exercise testing outcomes depending on indication for heart transplantation. We aimed to determine whether the number of pretransplant sternotomies is associated with differences in heart rate response during exercise testing.MethodsA retrospective analysis of our institutional pediatric heart transplant data between 2004 and 2022 was performed. Patients were categorized by indication for transplantation into a cardiomyopathy (CM) group if they had a congenital or acquired cardiomyopathy or a congenital heart disease (CHD) group including all other forms of congenital cardiac anatomic abnormalities.ResultsCHD patients (<jats:italic>n</jats:italic> = 40) differed from CM patients (<jats:italic>n</jats:italic> = 53) by mean number of sternotomies prior to transplant (2.4 ± 1.8 vs. 0.5 ± 0.9, <jats:italic>p</jats:italic> &lt; 0.001). There were no significant differences in echocardiographic function or catheterization hemodynamics. In cardiopulmonary exercise testing performance, the congenital heart disease group had a significantly higher resting heart rate (91.8 ± 11.2 vs. 86.4 ± 10.2 bpm, <jats:italic>p</jats:italic> = 0.019), lower percent predicted age‐predicted maximal heart rate achieved (78.3 ± 8.5% vs. 83.2 ± 11.4%, <jats:italic>p</jats:italic> = 0.032), and lower heart rate reserve (68.6 ± 19.8 vs. 84.4 ± 24.0 bpm, <jats:italic>p</jats:italic> = 0.001) despite a similar age and average time from transplantation. Regression analysis confirmed number of pretransplant sternotomies as a main predictor of heart rate metrics.ConclusionsThere is greater chronotropic incompetence in patients who underwent transplantation due to congenital heart disease compared to cardiomyopathy. The groups differ significantly by number of sternotomies, potentially supporting the hypothesis that prior surgical disruption of cardiac innervation may cause decreased chronotropic response to exercise following transplantation.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142259770","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
De Novo and Progressive Pulmonary Vein Stenosis Following Pediatric Heart Transplantation: A Multicenter Retrospective Study. 小儿心脏移植术后新发和进行性肺静脉狭窄:一项多中心回顾性研究
IF 1.2 4区 医学
Pediatric Transplantation Pub Date : 2024-08-01 DOI: 10.1111/petr.14828
Arene Butto, Conor O'Halloran, James Kuo, Anna Joong, Amanda L Hauck, Alan Nugent, William Mahle, Paul Tannous
{"title":"De Novo and Progressive Pulmonary Vein Stenosis Following Pediatric Heart Transplantation: A Multicenter Retrospective Study.","authors":"Arene Butto, Conor O'Halloran, James Kuo, Anna Joong, Amanda L Hauck, Alan Nugent, William Mahle, Paul Tannous","doi":"10.1111/petr.14828","DOIUrl":"10.1111/petr.14828","url":null,"abstract":"<p><strong>Background: </strong>Pulmonary vein stenosis (PVS) is a rare condition in which neointimal proliferation leads to venous and arterial hypertension. Little is known about PVS after heart transplant (HTx) in children. We sought to describe the characteristics and outcomes of children who develop PVS after HTx.</p><p><strong>Methods: </strong>We performed a retrospective review of patients ≤18 years old who underwent HTx at two HTx centers between April 2012 and October 2023. Patients with PVS were identified via database queries. Cardiac diagnosis, PVS location and extent, and outcomes were recorded.</p><p><strong>Results: </strong>Over 11.5 years, 422 patients underwent HTx across both centers. Nineteen patients with PVS (10 male) were identified, 15 with de novo PVS. Sixteen had underlying congenital heart disease (CHD), two with anomalous pulmonary venous return. PVS was diagnosed at a median of 2 months (range 2 weeks to 14 years) after HTx. At time of initial diagnosis, 13 patients had one-vessel PVS. At final follow-up, 7/19 (37%) had increases in the number of vessels involved. Six patients underwent surgery, and nine patients had stent or balloon angioplasty. Two patients were treated for pulmonary hypertension following PVS diagnosis. Three patients died from right heart failure secondary to PVS.</p><p><strong>Conclusions: </strong>This is the largest study to describe the characteristics of post-HTx PVS in children. PVS occurs in 4.5% of HTx, and underlying CHD is a strong risk factor. Multiple vessels can be involved and may require catheter-based or surgical intervention. Clinicians must be vigilant in monitoring the development of PVS in this population.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141731390","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
Norovirus Management and Outcomes in a Multicenter Pediatric Kidney Transplant Population. 多中心小儿肾移植人群中的诺如病毒管理和结果。
IF 1.2 4区 医学
Pediatric Transplantation Pub Date : 2024-08-01 DOI: 10.1111/petr.14821
Rachel M Engen, Michelle Keyser, Ziou Jiang, Sarah Kizilbash
{"title":"Norovirus Management and Outcomes in a Multicenter Pediatric Kidney Transplant Population.","authors":"Rachel M Engen, Michelle Keyser, Ziou Jiang, Sarah Kizilbash","doi":"10.1111/petr.14821","DOIUrl":"10.1111/petr.14821","url":null,"abstract":"<p><strong>Background: </strong>Norovirus is the most common cause of viral gastroenteritis. Studies in adult kidney recipients have documented significant morbidity associated with norovirus infection, but there are few studies in pediatric recipients.</p><p><strong>Methods: </strong>Multicenter retrospective cohort study of pediatric kidney transplant recipients with norovirus, confirmed by stool PCR, between January 1, 2008, and December 31, 2018. Outcomes of interest included duration of diarrhea, incidence of chronic diarrhea, management strategies, and graft function.</p><p><strong>Results: </strong>Forty pediatric kidney transplant recipients from four centers were identified for inclusion. Median age at transplant was 5.4 years (IQR 2.2-11.2 years), and median time post-transplant was 1.9 years (IQR 0.8-3.8 years). Median diarrheal duration was 16 days (IQR 6.0-41.5 days); 15 patients (43%) had acute diarrhea, 8 (23%) had persistent, and 12 (30%) had chronic diarrhea. Twenty-one (53%) patients developed acute kidney injury. Thirty-five (88%) patients required supplemental fluids, 8 (20%) patients underwent immunosuppression reduction for a median of 22 days, 5 (13%) were treated with nitazoxanide, and 5 (13%) received oral immunoglobulin. Acute rejection was diagnosed in 3 (8%) patients within 6 months of norovirus diagnosis. We observed no sustained decline in eGFR at 12 months after diarrhea resolution (median eGFR difference: 2.8 mL/min/1.73 m<sup>2</sup> [IQR: -17.1, 7.4]). Of the patients in the cohort, two lost their graft at 6.8 and 30.0 months after the onset of diarrhea.</p><p><strong>Conclusion: </strong>Norovirus is associated with significant morbidity in pediatric kidney transplant recipients. Various treatment interventions are being employed for norovirus infection. Larger studies, both observational and interventional, are needed to determine the optimal treatment.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141591019","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
Safety and infectious outcomes in pediatric kidney transplant recipients after COVID-19 vaccination: A pediatric nephrology research consortium study. 小儿肾移植受者接种 COVID-19 疫苗后的安全性和感染结果:儿科肾脏病研究联盟的一项研究。
IF 1.2 4区 医学
Pediatric Transplantation Pub Date : 2024-06-01 DOI: 10.1111/petr.14786
Travis Churilla, Clarkson Crane, Rajasree Sreedharan, Bakri J Alzarka, Olga Charnaya, Namrata G Jain, Helen Pizzo, Asifhusen Mansuri, Amrish Jain, Manpreet Grewal, Joseph D Fishbein, Alexander J Kula, Taylor Heald-Sargent, Debora Matossian, Priya S Verghese
{"title":"Safety and infectious outcomes in pediatric kidney transplant recipients after COVID-19 vaccination: A pediatric nephrology research consortium study.","authors":"Travis Churilla, Clarkson Crane, Rajasree Sreedharan, Bakri J Alzarka, Olga Charnaya, Namrata G Jain, Helen Pizzo, Asifhusen Mansuri, Amrish Jain, Manpreet Grewal, Joseph D Fishbein, Alexander J Kula, Taylor Heald-Sargent, Debora Matossian, Priya S Verghese","doi":"10.1111/petr.14786","DOIUrl":"10.1111/petr.14786","url":null,"abstract":"<p><strong>Background: </strong>Adult kidney transplant recipients (KTRs) fully vaccinated against COVID-19 have substantial morbidity and mortality related to SARS-CoV-2 infection compared with the general population. However, little is known regarding the safety and efficacy of the COVID-19 vaccination series in pediatric KTRs.</p><p><strong>Methods: </strong>A multicenter, retrospective observational study was performed across nine pediatric transplantation centers. Eligible KTRs fully vaccinated against COVID-19 were enrolled and data were collected pertaining to SARS-CoV-2 infection incidence and severity, graft outcomes and post-vaccination safety profile, as well as overall patient survival.</p><p><strong>Results: </strong>A total of 247 patients were included in this investigation with a median age at transplantation of 11 years (IQR 5-15). SARS-CoV-2 infection was observed in 30/110 (27.27%) of fully vaccinated patients, tested post-transplant, within the defined follow-up period. Of these patients, 6/30 (18.18%) required hospitalization and 3/30 (12.12%) required reduction in immunosuppression, with no reported deaths. De novo donor-specific antibodies (DSAs) were found in 8/86 (9.30%) of DSA-tested patients with two experiencing rejection and subsequent graft loss. The overall incidence of rejection and graft loss among the total cohort was 11/247 (4.45%) and 6/247 (3.64%), respectively. A 100% patient survival was observed.</p><p><strong>Conclusions: </strong>Observationally, infectious outcomes of SARS-CoV-2 in fully vaccinated pediatric KTRs are excellent, with a low incidence of infection requiring hospitalization and no associated deaths. Though de novo DSAs were observed, there was minimal graft rejection and graft loss reported in the total cohort.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141065978","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
Calcineurin inhibitor-related hyperkalemia is caused by hyporeninemic hypoaldosteronism and fludrocortisone is an effective treatment: Report of a case series and review of the literature. 钙神经蛋白抑制剂相关高钾血症是由胰岛素分泌过少引起的,氟氢可的松是一种有效的治疗方法:系列病例报告和文献综述。
IF 1.3 4区 医学
Pediatric Transplantation Pub Date : 2024-06-01 DOI: 10.1111/petr.14778
Yagmur Unsal, Demet Baltu, Bora Gulhan, Fatma Visal Okur, Fatih Ozaltın, Ali Düzova, Rezan Topaloğlu, Zeynep Alev Ozon, Elmas Nazlı Gonç
{"title":"Calcineurin inhibitor-related hyperkalemia is caused by hyporeninemic hypoaldosteronism and fludrocortisone is an effective treatment: Report of a case series and review of the literature.","authors":"Yagmur Unsal, Demet Baltu, Bora Gulhan, Fatma Visal Okur, Fatih Ozaltın, Ali Düzova, Rezan Topaloğlu, Zeynep Alev Ozon, Elmas Nazlı Gonç","doi":"10.1111/petr.14778","DOIUrl":"10.1111/petr.14778","url":null,"abstract":"<p><strong>Introduction: </strong>Calcineurin inhibitors (CNIs) are widely used in transplantation. Although CNI-related hyperkalemia is common (10%-60.6%), the underlying pathogenetic mechanism is not well-elucidated and may lead to dose adjustment or treatment withdrawal.</p><p><strong>Objective: </strong>The aim of this study is to describe CNI-related hyperkalemia due to hyporeninemic hypoaldosteronism in pediatric transplant recipients who were successfully treated with fludrocortisone.</p><p><strong>Method: </strong>In a total of 55 hematopoietic stem cell (HSCT) and 35 kidney transplant recipients followed according to institutional immunosuppression protocols, recipients diagnosed with CNI-related hyperkalemia were reviewed. Recipients who were receiving intravenous fluid, potassium, or were diagnosed with hemolysis, acute graft rejection, or had an eGFR < 30 mL/min/1.73m<sup>2</sup>, were excluded. A detailed analysis of clinical history as well as biochemical studies was carried out to reveal possible pathophysiology.</p><p><strong>Results: </strong>Three pediatric transplant recipients (one HSCT, two kidney transplantation) with findings of hyperkalemia, hyponatremia, and a mild elevation in blood urea nitrogen while on CNIs were recruited. Urinary potassium excretion was diminished while sodium excretion was increased. Plasma aldosterone levels were low, and renin was not increased in response. Primary adrenal insufficiency was ruled out, and hyporeninemic hypoaldosteronism was diagnosed. CNI-related hyperkalemia was detected earlier in case 1, who had HSCT (22 days), than in the second and third cases, who had kidney transplantation (24 and 30 months post-transplantation, respectively). The discrepancy was hypothesized to be explained by higher overall CNI dose due to higher serum target CNI used in HSCT than kidney transplantation. Electrolyte imbalance was reversed upon administration of physiologic dose fludrocortisone (0.05 mg, daily), while fludrocortisone was ceased after CNI withdrawal in case 1, which is additional evidence for the etiological association of CNIs and hyporeninemic hypoaldosteronism.</p><p><strong>Conclusion: </strong>Our three cases strengthen the premise that CNI-related hyperkalemia may be due to hyporeninemic hypoaldosteronism, and the timing and severity may be related to CNI dose. Fludrocortisone is a safe and effective treatment in CNI-related hyperkalemia, providing maintenance of CNIs, which are one of the essential therapeutic agents for pediatric transplantation.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141066039","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
The role of donor type and pre-transplant immunosuppression on outcomes of hematopoietic stem cell transplantation in children and young adults with severe aplastic anemia. 供体类型和移植前免疫抑制对重型再生障碍性贫血儿童和青少年造血干细胞移植结果的影响。
IF 1.2 4区 医学
Pediatric Transplantation Pub Date : 2024-06-01 DOI: 10.1111/petr.14784
Reema Kashif, Biljana Horn, Jordan Milner, Michael Joyce, Mansi Dalal, Jin-Ju Lee, Kevin McNerney, Jessica Cline, John Fort, Paul Castillo, Jorge Galvez, Warren Alperstein, John Ligon, Edward Ziga, David Crawford, Deepak Chellapandian
{"title":"The role of donor type and pre-transplant immunosuppression on outcomes of hematopoietic stem cell transplantation in children and young adults with severe aplastic anemia.","authors":"Reema Kashif, Biljana Horn, Jordan Milner, Michael Joyce, Mansi Dalal, Jin-Ju Lee, Kevin McNerney, Jessica Cline, John Fort, Paul Castillo, Jorge Galvez, Warren Alperstein, John Ligon, Edward Ziga, David Crawford, Deepak Chellapandian","doi":"10.1111/petr.14784","DOIUrl":"10.1111/petr.14784","url":null,"abstract":"<p><strong>Background: </strong>The goal of this study was to assess the effect of donor type and pre-transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA).</p><p><strong>Methods: </strong>This retrospective, multi-center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first- or second-line treatment.</p><p><strong>Results: </strong>The median age at HSCT for all 52 patients was 15 years (range 1-25). The 3-year overall survival (OS) by donor type were as follows: 95% [95% CI 85.4-99] for matched related donors (MRD) (N = 24), 84% [95% CI 63.5-99] for haploidentical (N = 13), and 71% [95% CI 36-99] for matched unrelated donors (MUD) (N = 7). The 3-year OS was 81% [95% CI 69.7-99] for all patients, 90.5% [95% CI 79.5-99] for non-IST patients (N = 27), and 70% [95% CI 51-99] for IST patients (N = 24) (log-rank p = .04). Survival of haploidentical HSCT (haplo-HSCT) recipients with post-transplant cyclophosphamide (PTCy) (N = 13) was excellent for both groups: 100% for non-IST patients (N = 3) and 80% for IST patients (N = 10). The 3-year OS for patients with previous IST by donor type in groups where >5 patients were available was 78.8% [95% CI 52.3-99] for haplo-HSCT (N = 10) and 66.7% [95% CI 28.7-99] for MUD (N = 6). Although it appears that patients receiving HSCT ≥6 months after the start of IST had worse survival, the number of patients in each category was small and log-rank was not significant(p = .65).</p><p><strong>Conclusions: </strong>Patients receiving MUD and haplo-HSCT with PTCy had similar outcomes, suggesting that haplo-HSCT with PTCy could be included in randomized trials of upfront IST versus alternative donor HSCT.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141066012","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
Comparison of cystatin C-based estimated glomerular filtration rate with measured glomerular filtration rate in a pediatric cohort of patients with chronic kidney disease. 基于胱抑素 C 的估算肾小球滤过率与测量肾小球滤过率在儿科慢性肾病患者队列中的比较。
IF 1.2 4区 医学
Pediatric Transplantation Pub Date : 2024-06-01 DOI: 10.1111/petr.14776
Tilde Ostendorf Lindvig, Jane Angel Simonsen, Oke Gerke, Helle Charlotte Thiesson
{"title":"Comparison of cystatin C-based estimated glomerular filtration rate with measured glomerular filtration rate in a pediatric cohort of patients with chronic kidney disease.","authors":"Tilde Ostendorf Lindvig, Jane Angel Simonsen, Oke Gerke, Helle Charlotte Thiesson","doi":"10.1111/petr.14776","DOIUrl":"10.1111/petr.14776","url":null,"abstract":"<p><strong>Background: </strong>It is essential to have an accurate assessment of the renal function of patients with chronic kidney disease to monitor, treat, and predict further development of the condition. Measurement of renal function in terms of glomerular filtration rate (GFR) requires either urine or blood sampling, but especially in children, more simple methods of measurement are preferable. The main objective of this study was to examine if the estimated GFR (eGFR) calculated with different cystatin-C-based equations was comparable to the GFR measured by a radiotracer (mGFR) in pediatric patients.</p><p><strong>Methods: </strong>In this retrospective study, 28 pediatric patients contributed with 73 pairs of measurements collected within 5 years. Bland-Altman Limits of Agreement were used to evaluate the performance and accuracy of two different cystatin-C-based estimates, the CKiD<sub>Crea-CysC</sub> and the CKiD<sub>U25</sub> respectively, compared to an mGFR based on plasma clearance of technetium-99m-diethylenetriaminepentaacetic acid or chromium-51-ethylenediaminetetraacetic acid.</p><p><strong>Results: </strong>Using the CKiD<sub>Crea-CysC</sub> equation, 58.9% of the datasets were within P10 and 87.7% were within P30. The mean difference was 4.8 mL/min/1.73m<sup>2</sup> (standard deviation: 8.5 mL/min/1.73m<sup>2</sup>) and tended to overestimate GFR and thereby overrate the kidney function within the entire GFR range. Using the CKiD<sub>U25</sub> equation, 53.4% were within P10 and 93.2% within P30. The mean difference was -2.9 mL/min/1.73m<sup>2</sup> (standard deviation: 8.4 mL/min/1.73m<sup>2</sup>), but the difference varied with the GFR value.</p><p><strong>Conclusions: </strong>A cystatin-C-based eGFR provides a viable substitute for monitoring renal function in pediatric patients with chronic kidney disease. However, it has a lower accuracy than mGFR and can therefore not replace mGFR in clinical use.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141081988","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 from hematopoietic stem cell transplantation following treosulfan-based conditioning: A clinical and pharmacokinetic analysis. 以曲硫安为基础的调理后造血干细胞移植的结果:临床和药代动力学分析
IF 1.2 4区 医学
Pediatric Transplantation Pub Date : 2024-06-01 DOI: 10.1111/petr.14780
Sebastian P A Rosser, Alice Brewer, Melissa Gabriel, Melanie Wong, Jason Chung, Andrew J McLachlan, Christa E Nath, Steven J Keogh, Peter J Shaw
{"title":"Outcomes from hematopoietic stem cell transplantation following treosulfan-based conditioning: A clinical and pharmacokinetic analysis.","authors":"Sebastian P A Rosser, Alice Brewer, Melissa Gabriel, Melanie Wong, Jason Chung, Andrew J McLachlan, Christa E Nath, Steven J Keogh, Peter J Shaw","doi":"10.1111/petr.14780","DOIUrl":"10.1111/petr.14780","url":null,"abstract":"<p><strong>Background: </strong>The aims of this study are to report our experience with treosulfan-based conditioning regimens for patients with non-malignant hematologic conditions, correlating clinical outcomes at different time points post-transplant with treosulfan exposure (AUC).</p><p><strong>Methods: </strong>This study was a single-center observational study investigating overall survival (OS), disease-free survival (DFS), and event-free survival (EFS) end-points post-transplant. The consequences of treosulfan AUC with respect to toxicity, correction of underlying disease, and long-term chimerism were also explored using pharmacokinetic analysis.</p><p><strong>Results: </strong>Forty-six patients received 49 transplants with treosulfan and fludarabine-based conditioning between 2005 and 2023. Twenty-four patients also received thiotepa. Donor chimerism was assessed on either whole blood or sorted cell lines at different time points post-transplant. Thirty-nine patients received treosulfan pharmacokinetic assessment to evaluate cumulative AUC, with five infants receiving real-time assessment to facilitate daily dose adjustment. OS, DFS, and EFS were 87%, 81%, and 69%, respectively. Median follow-up was 32.1 months (range 0.82-160 months) following transplant. Lower EFS was associated with patient age (<1 year; p = .057) and lower cumulative treosulfan dose (<42 g/m<sup>2</sup>; p = .003). Stable donor chimerism in B-cell, NK-cell, and granulocyte lineages at 1-year post-transplant were more prevalent in patients receiving thiotepa conditioning. Two infants required daily dose adjustment to treosulfan to avoid high AUC.</p><p><strong>Conclusions: </strong>Excellent clinical outcomes and stable chimerism were observed in this patient series. The addition of thiotepa conferred no significant toxicity and trended toward sustained ongoing donor engraftment. Correlating treosulfan AUC with long-term patient outcomes is required.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141065975","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
Effect of body mass index on exercise capacity following pediatric heart transplantation 体重指数对小儿心脏移植后运动能力的影响
IF 1.3 4区 医学
Pediatric Transplantation Pub Date : 2024-05-04 DOI: 10.1111/petr.14772
Alan P. Wang, Kendra Ward, Garett Griffith, Katheryn Gambetta
{"title":"Effect of body mass index on exercise capacity following pediatric heart transplantation","authors":"Alan P. Wang, Kendra Ward, Garett Griffith, Katheryn Gambetta","doi":"10.1111/petr.14772","DOIUrl":"https://doi.org/10.1111/petr.14772","url":null,"abstract":"BackgroundObesity and impaired exercise tolerance following heart transplantation increase the risk of post‐transplant morbidity and mortality. The aim of this study was to evaluate the effect of body mass index on markers of exercise capacity in pediatric heart transplant recipients and compare this effect with a healthy pediatric cohort.MethodsA retrospective analysis of cardiopulmonary exercise test data between 2004 and 2022 was performed. All patients exercised on a treadmill using the Bruce protocol. Inclusion criteria included patients aged 6–21 years, history of heart transplantation (transplant cohort) or no cardiac diagnosis (control cohort) at the time of testing, and a maximal effort test. Patients were further stratified within these two cohorts as underweight, normal, overweight, and obese based on body mass index groups. Two‐way analyses of variance were performed with diagnosis and body mass index category as the independent variables.ResultsA total of 250 exercise tests following heart transplant and 1963 exercise tests of healthy patients were included. Heart transplant patients across all body mass index groups had higher resting heart rate and lower maximal heart rate, heart rate recovery at 1 min, exercise duration, and peak aerobic capacity (VO<jats:sub>2peak</jats:sub>). Heart transplant patients in the normal and overweight body mass index categories had higher VO<jats:sub>2peak</jats:sub> and exercise duration when compared to underweight and obese patients.ConclusionUnderweight status and obesity are strongly associated with lower VO<jats:sub>2peak</jats:sub> and exercise duration in heart transplant patients. Normal and overweight heart transplant patients had the best markers of exercise capacity.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140836889","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
The use of once‐daily LCP‐Tacrolimus with adolescent and young adult solid organ transplant recipients 对青少年和年轻成人实体器官移植受者使用每日一次的 LCP-Tacrolimus
IF 1.3 4区 医学
Pediatric Transplantation Pub Date : 2024-05-04 DOI: 10.1111/petr.14777
Sarah Householder, Adarsh Ramakrishnan, Justin K. Chen, Lindsey Gorsch, Demetra Tsapepas, Steven Lobritto, Anna Rundle, Jennifer M. Vittorio
{"title":"The use of once‐daily LCP‐Tacrolimus with adolescent and young adult solid organ transplant recipients","authors":"Sarah Householder, Adarsh Ramakrishnan, Justin K. Chen, Lindsey Gorsch, Demetra Tsapepas, Steven Lobritto, Anna Rundle, Jennifer M. Vittorio","doi":"10.1111/petr.14777","DOIUrl":"https://doi.org/10.1111/petr.14777","url":null,"abstract":"BackgroundAdolescent and young adult (AYA) solid organ transplant (SOT) recipients experience increased rates of rejection and graft loss surrounding the time of health care transition, in part due to poor medication adherence. This study aims to examine the impact of a once‐daily formulation of tacrolimus, LCP‐tacrolimus (LCPT), on medication adherence for AYA SOT patients.MethodsA retrospective descriptive analysis was performed for all patients who underwent SOT and were prescribed LCPT after the age of 12 at our single‐center pediatric hospital. Medication adherence was assessed via provider documentation and the medication level variability index (MLVI).ResultsTwenty‐nine patients were prescribed LCPT as part of their immunosuppression regimen. Twenty patients were converted to LCPT from immediate‐acting (IR) tacrolimus; six patients were initiated immediately following transplant, and three patients were unable to receive LCPT due to insurance denial. There was a numeric improvement in medication adherence for converted patients when measured by provider assessment (45.0% vs. 68.4%, <jats:italic>p</jats:italic> = .140) and MLVI (40.0% vs. 71.4%, <jats:italic>p</jats:italic> = .276), though these did not reach statistical significance. There were no differences in episodes of rejection or adverse effects. LCPT prescription was not associated with decreased medication burden, and two patients transitioned back to IR tacrolimus due to increased cost.ConclusionsLCPT use did not significantly improve patient adherence; however, it resulted in numerically higher perceived and measured adherence rates. LCPT appears to be safe and effective in the management of SOT recipients; however, it may not affect pill burden and may result in a higher financial burden. Use may be considered for a select group of AYA SOT recipients.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":null,"pages":null},"PeriodicalIF":1.3,"publicationDate":"2024-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140836890","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
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