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1.
A strategy to improve treatment‐related mortality and abandonment of therapy for childhood ALL in a developing country reveals the impact of treatment delays
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Amaranto Suarez MD Martha Piña MD Diana X. Nichols‐Vinueza MD John Lopera MD Lyda Rengifo MD Mauricio Mesa MD Marcela Cardenas RN Lisa Morrissey RN Galo Veintemilla MD Martha Vizcaino MD Ligia Del Toro MD Victor Vicuna PhD Jorge Fernandez LICSW Donna Neuberg ScD Kristen Stevenson MS Alejandro Gutierrez MD 《Pediatric blood & cancer》2015,62(8):1395-1402
Background
Treatment‐related mortality and abandonment of therapy are major barriers to successful treatment of childhood acute lymphoblastic leukemia (ALL) in the developing world.Procedure
A collaboration was undertaken between Instituto Nacional de Cancerologia (Bogota, Colombia), which serves a poor patient population in an upper‐middle income country, and Dana‐Farber/Boston Children's Cancer and Blood Disorders Center (Boston, USA). Several interventions aimed at reducing toxic deaths and abandonment were implemented, including a reduced‐intensity treatment regimen and a psychosocial effort targeting abandonment. We performed a cohort study to assess impact.Results
The Study Population comprised 99 children with ALL diagnosed between 2007 and 2010, and the Historic Cohort comprised 181 children treated prior to the study interventions (1995–2004). Significant improvements were achieved in the rate of deaths in complete remission (13% to 3%; P = 0.005), abandonment (32% to 9%; P < 0.001), and event‐free survival with abandonment considered an event (47% to 65% at 2 years; P = 0.016). However, relapse rate did not improve. Medically unnecessary treatment delays were common, and landmark analysis revealed that initiating the PIII phase of therapy ≥4 weeks delayed predicted markedly inferior disease‐free survival (P = 0.016). Conversely, patients who received therapy without excessive delays had outcomes approaching those achieved in high‐income countries.Conclusions
Implementation of a twinning program was followed by reductions in abandonment and toxic deaths, but relapse rate did not improve. Inappropriate treatment delays were common and strongly predicted treatment failure. These findings highlight the importance of adherence to treatment schedule for effective therapy of ALL. Pediatr Blood Cancer 2015;62:1395–1402. © 2015 Wiley Periodicals, Inc. 相似文献2.
SIOP‐PODC adapted risk stratification and treatment guidelines: Recommendations for neuroblastoma in low‐ and middle‐income settings
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Nehal S. Parikh MD Scott C. Howard MD Guillermo Chantada MD Trijn Israels MD Mohammed Khattab MD Patricia Alcasabas MD Catherine G. Lam MD Lawrence Faulkner MD Julie R. Park MD Wendy B. London PhD Katherine K. Matthay MD 《Pediatric blood & cancer》2015,62(8):1305-1316
3.
A Framework for Adapted Nutritional Therapy for Children With Cancer in Low‐ and Middle‐Income Countries: A Report From the SIOP PODC Nutrition Working Group
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Elena J. Ladas PhD RD Brijesh Arora MD DM Scott C. Howard MD Paul C. Rogers MD Terezie T. Mosby EdD RD Ronald D. Barr MB ChB MD 《Pediatric blood & cancer》2016,63(8):1339-1348
The utilization of adapted regimens for the treatment of pediatric malignancies has greatly improved clinical outcomes for children receiving treatment in low‐ and middle‐income countries (LMIC). Nutritional depletion has been associated with poorer outcomes, increased abandonment of therapy, and treatment‐related toxicities. Surveys have found that nutritional intervention is not incorporated routinely into supportive care regimens. Establishing nutritional programs based upon institutional resources may facilitate the incorporation of nutritional therapy into clinical care in a way that is feasible in all settings. We present a framework for establishing and monitoring of nutritional care based on the infrastructure of institutions in LMIC. 相似文献
4.
Ashleigh Kussman David Shyr Gregory Hale Benjamin Oshrine Aleksandra Petrovic 《Pediatric blood & cancer》2019,66(1)
Relapse remains the most common cause of treatment failure after hematopoietic cell transplantation for acute myeloid leukemia. Inability to achieve hematologic complete remission has been a barrier to transplant for patients with refractory disease. We describe six children with refractory myeloid disease undergoing transplant in chemotherapy‐induced aplasia, as a strategy to facilitate curative therapy in refractory patients. Clofarabine‐ or high‐dose cytarabine‐based chemotherapy regimens were used to achieve marrow aplasia, followed by reduced‐intensity conditioning and allogeneic transplant before hematologic recovery. Long‐term disease control was achieved in five, with one transplant‐related mortality, suggesting the feasibility of this approach. 相似文献
5.
Long‐term outcomes of simultaneous heart and kidney transplantation in pediatric recipients
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Patricia L. Weng Juan Carlos Alejos Nancy Halnon Qiuheng Zhang Elaine F. Reed Eileen Tsai Chambers 《Pediatric transplantation》2017,21(7)
Pediatric sHKTx has become an effective therapy for patients with combined cardiac and renal failure. Often, these patients develop human leukocyte antigen antibodies from their previous allografts and are therefore more difficult to re‐transplant. We describe the largest case series of a predominantly sensitized pediatric sHKTx with emphasis on medical management and patient outcomes. Demographics, clinical characteristics, antibody, and biopsy data were retrospectively collected from University of California, Los Angeles database and correlated with short‐ and long‐term patient and allograft outcomes of all sHKTx performed between 2002 and 2015. We identified seven pediatric patients who underwent sHKTx at our center. Mean age at time of sHKTx was 13.7 years and 85.7% were re‐graft patients. 57.1% were sensitized with cPRA >50% and another 57.1% had preformed donor‐specific antibody. Five‐year renal allograft survival and patient survival was 85.7% for both end‐points. The remaining six patients are all alive (mean follow‐up 78.5 months) with good kidney and heart function. sHKTx in a population with increased immunological risk can be associated with good long‐term outcomes and offers potential guidance to the pediatric transplant community where data are limited. 相似文献
6.
Trijn Israels Dalida Pidini Eric Borgstein Simon Bailey Clara Tump George Chagaluka 《Pediatric hematology and oncology》2013,30(3):196-202
AbstractWilms tumor (WT) has a survival rate above 90% in high income countries. Reported survival rates in sub-Saharan Africa are much lower and long-term outcome is not well known as follow-up is challenging. In Blantyre, Malawi, an adapted WT treatment guideline with preoperative chemotherapy, supportive care, and strategies to enable children and parents to complete treatment was introduced in 2006. Between 2006 and 2011, 73 children with a unilateral WT were treated. Follow-up, including home visits when needed, was done. Median follow-up time is 5 years (range 14–95 months). Two and five-year event free survivals are 46 and 42%. Causes of treatment failure are: 7% (5/73) abandonment of treatment, 15% (11/73) death during treatment and 30% (22/73) disease-related deaths (persistent disease and relapse). Long-term follow-up is challenging but necessary to be able to assess outcome and the true impact of interventions. 相似文献
7.
Yu JJ 《Korean journal of pediatrics》2012,55(3):83-87
Several authors suggested that the clinical characteristics of incomplete presentation of Kawasaki disease are similar to those of complete presentation and that the 2 forms of presentation are not separate entities. Based on this suggestion, a diagnosis of incomplete Kawasaki disease in analogy to the findings of complete presentation is reasonable. Currently, the diagnosis of incomplete Kawasaki disease might be made in cases with fewer classical diagnostic criteria and with several compatible clinical, laboratory or echocardiographic findings on the exclusion of other febrile illness. Definition of incomplete presentation in which coronary artery abnormalities are included as a necessary condition, is restrictive and specific. The validity of the diagnostic criteria of incomplete presentation by the American Heart Association should be thoroughly tested in the immediate future. 相似文献
8.
Agardh D Nilsson A Tuomi T Lindberg B Carlsson AK Lernmark A Ivarsson SA 《Pediatric diabetes》2001,2(2):58-65
Abstract: Aims: The aims were to estimate the diagnostic sensitivity and specificity of autoantibodies to tissue transglutaminase (IgA‐ and IgG‐tTG), gliadin (AGA) and endomysium (EMA) in relation to human leukocyte antigen (HLA)‐DQB1 alleles to identify silent celiac disease at diagnosis of type 1 diabetes. Methods: IgA‐ and IgG‐tTG were measured in radioligand binding assays in 165 type 1 diabetic patients. Data on HLA‐DQB1 were available for 148 patients and on both AGA and EMA for 164 patients. For patients considered positive for AGA or EMA, or both, an intestinal biopsy was suggested. HLA‐DQB1 typing was carried out by polymerase chain reaction and hybridization with allele specific probes. Results: Three patients, left out from further study of antibodies, but not from HLA‐DQB1 analysis, had treated celiac disease at diagnosis. Out of the other 162 type 1 diabetic patients tested, nine had IgA‐tTG, six IgG‐tTG, eight EMA, and 11 AGA. Biopsy was suggested for nine patients, of whom six showed villous atrophy, one did not and two refused to participate. Thus, silent celiac disease was probable in 8/162 and biopsy‐verified in 6/162, where five patients were AGA‐positive and six either EMA‐, IgA‐tTG‐ or IgG‐tTG‐positive. Of the 11 patients with celiac disease (three with treated and eight with silent celiac disease), 10 were HLA‐DQB1‐typed, of whom 65% (13/20) had the DQB1*02 allele, compared with 36% (100/276; p = 0.011) of those without celiac disease. IgA‐tTG levels were higher in patients having either *02 or *0302 (0.6; ?1.3–112.4 RU) compared with those not having these alleles (0.4; ?0.7–3.4 RU; p = 0.023). Conclusion: IgA‐tTG are HLA‐DQB1*02‐associated autoantibodies with high sensitivity and specificity for silent celiac disease at diagnosis of type 1 diabetes. 相似文献
9.
Chen‐Ming Huang Sheng‐Hong Tseng Chia‐Chee Weng Yun Chen 《Pediatric transplantation》2013,17(1):E4-E8
MMIHS is a rare congenital disease. It is characterized by distended urinary bladder, small colon and intestinal hypoperistalsis, or aperistalsis with normal morphology. There is no specific treatment for MMIHS, and most patients have to be maintained by TPN, which frequently causes TPN‐related liver failure, loss of venous access, or catheter‐related sepsis. The prognosis of patients with MMIHS is poor, and most patients die early. Multivisceral transplantation including stomach, duodenum, intestine, and liver has been used for the treatment of patients with MMIHS because these patients often have liver failure. We report an eight‐yr‐old patient with MMIHS who was treated with isolated intestinal transplantation. She had completely oral intake during the four yr of follow‐up. The experience in this case suggests isolated intestinal transplantation may be indicated in selected cases with MMIHS. 相似文献
10.
Sudo D Monobe Y Yashiro M Mieno MN Uehara R Tsuchiya K Sonobe T Nakamura Y 《European journal of pediatrics》2012,171(4):651-656
Incomplete Kawasaki disease (KD) is associated with delayed diagnosis and treatment, which in turn can lead to the development
of coronary artery lesions (CALs). The aim of this study was to determine the epidemiological features of incomplete KD compared
with complete KD and to identify risk factors for CALs from incomplete KD patients using data from a nationwide survey of
2007–2008 in Japan. A total of 23,263 patients were classified according to the number of principal clinical signs: 80% (n = 18,620) had complete forms of KD, 14.2% had four principal signs, 4.6% had three signs, and 1.2% had only one or two signs.
In comparison with complete KD cases, the prevalence of CAL development tended to be larger and the proportion receiving initial
intravenous immunoglobulin (IVIG) treatment were significantly smaller in patients with incomplete forms. In addition, hospital
attendance after 7 days of illness or later was significantly associated with CAL development in all incomplete groups (OR:
2.52 in total patients with incomplete KD, 3.26 in those with one or two principal signs, 2.94 in those with three signs,
2.35 in those with four signs). Conclusion The higher prevalence of CALs in incomplete KD reflects difficulties in diagnosis and delays in treatment. More timely diagnosis
and treatment of incomplete KD patients could further prevent the development of cardiac lesions. 相似文献
11.
川崎病(Kawasaki disease)是一种主要发生于5岁以下儿童的、以中小血管炎为主要病变的发热性疾病。虽然KD被报道已50余载,近年来对于KD的病因和病理研究也取得了实质性进展,但目前仍缺乏对KD早期诊断的特异性指标,尤其是对不完全性川崎病(incomplete Kawasaki disease,IKD)的诊断更加困难。目前对于IKD尚无明确的诊断标准,这导致临床上不能对IKD进行及时识别和规范治疗,从而导致冠状动脉病变的发生。因此,该文就IKD的概念、流行病学特点、诊断、治疗及随访管理进行总结,加深临床工作者对IKD的认识,希望有助于提高我国KD的临床诊治水平。 相似文献
12.
Sebastian O. Ekenze Okechukwu C. Okafor Akputa A. Obasi David C. Okafor Ikenna I. Nnabugwu 《Pediatric blood & cancer》2020,67(11)
We performed a systematic review to highlight trends in management and outcome of Wilms tumor (WT) in Africa in the past two decades (2000‐2019). Twenty‐seven studies involving 2250 patients were analyzed. Overall, barring regional variations, 57.7% of the cases presented with advanced disease, 57.3% completed planned treatment, and survival was 56.5%. The publications in the two decades did not show significant differences in proportions of cases with advanced disease, completion of treatment rate, and cases lost to follow up. However, significantly more cases received preoperative chemotherapy, and survival improved in the last decade (2010‐2019) compared to the earlier decade (2000‐2009). Survival of WT in Africa might have improved in the last decade, but challenges of delayed presentation and abandonment of treatment have persisted. Measures that will encourage early access to expert care as well as improve on treatment compliance may further improve survival of WT in Africa. 相似文献
13.
Increasing observation rates in low‐risk pediatric immune thrombocytopenia using a standardized clinical assessment and management plan (SCAMP®)
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Michelle L. Schoettler Dionne Graham Wen Tao Margaret Stack Elaine Shu Lauren Kerr Ellis J. Neufeld Rachael F. Grace 《Pediatric blood & cancer》2017,64(5)
An observational approach is recommended in newly diagnosed children with immune thrombocytopenia (ITP) at low risk of bleeding; however, there is no standard definition of risk. A standardized clinical assessment and management plan (SCAMP®), a modifiable practice guideline, was implemented and revised (SCAMP‐1 and SCAMP‐2) and applied to 71 newly diagnosed patients with ITP. The Buchanan and Adix bleeding score guided treatment and was modified by stratifying by low‐ and high‐risk grade 3 bleeding in SCAMP‐2. Observation rates increased from 40% to 74% from SCAMP‐1 to SCAMP‐2 (P < 0.05) with no bleeding complications. We propose a modified bleeding score that increased observation rates in low‐risk patients with ITP. 相似文献
14.
Hajime Hosoi 《Pediatrics international》2016,58(2):81-87
This article reviews the current status of treatment for children with rhabdomyosarcoma, according to the four risk groups. Low‐risk subgroup A: the Children's Oncology Group in the USA recently performed a clinical trial consisting of a chemotherapy regimen with a shortened treatment period and a reduced drug dosage. Patients in this group received only four cycles of vincristine and actinomycin D (VA) after four cycles of vincristine, actinomycin D, and cyclophosphamide (VAC) with cyclophosphamide (CPM) 1.2 g/m2 and their outcome was no worse than that obtained with previous regimens. Low‐risk subgroup B: although marked improvement in survival was seen with an intensive VAC regimen with CPM 2.2 g/m2/cycle (Intergroup Rhabdomyosarcoma Study [IRS]‐V, 1997–2004), the total dose of CPM in this regimen caused serious and fatal hepatic veno‐occlusive disease during treatment and probably cannot avoid infertility or possible secondary cancer as a late effect. Thereafter, a reduced‐dose regimen consisting of four cycles of VAC with CPM 1.2 g/m2 followed by 12 cycles of VA was investigated in the next study, but the outcome appeared to be worse than in IRS‐V. Intermediate‐risk group: no significant difference was found between VAC/vincristine, topotecan and cyclophispahamide (VTC) and intensive VAC in IRS‐V. The results of a subsequent regimen of VAC with CPM 1.2 g/m2 alternating with vincristine and irinotecan are awaited. High‐risk group: overall survival is approximately 30% and has not improved over the last 25 years. Although 18 month failure‐free survival (FFS) was improved with an intensive combination therapy regimen, 36 month FFS dropped to 32% and thus better novel approaches or additive treatments are needed. 相似文献
15.
Incomplete Kawasaki disease in a 12‐month‐old girl presenting with cardiac murmur and iron deficiency anemia
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Kawasaki disease (KD) is an acute necrotizing vasculitis that occurs in children <5 years of age. The cause of KD remains unknown, but various complications, including dilatation of the coronary arteries, can occur. Coronary artery aneurysm or ectasia are the most important complications of KD. Children with suspected KD who do not fulfill the diagnostic criteria may have incomplete KD. Given that incomplete KD is associated with delayed diagnosis and treatment, children with incomplete KD have a high risk of cardiovascular complications. Meanwhile, iron deficiency anemia (IDA) is one of the most prevalent micronutrient deficiencies in the world. Children with IDA are prone to infection and inflammation. We report the case of a 12‐month‐old girl with incomplete KD who presented with cardiac murmur and severe IDA. 相似文献
16.
Ruzanna Papyan Gevorg Tamamyan Samvel Danielyan Armen Tananyan Armen Muradyan Raya Saab 《Pediatric blood & cancer》2019,66(7)
We performed a literature review to examine barriers for rhabdomyosarcoma treatment in low‐resource settings, and identified 29 articles from 14 middle‐income countries, with none from low‐income countries. Notable findings included inconsistent use of local control modalities, lack of diagnostics in some settings, and high rate of abandonment specifically in low middle‐income countries. Reported limitations included lack of surgical expertise and/or radiation therapy, advanced stage of disease, and absence of health insurance. Although very poor outcomes were prevalent in several settings, good outcomes were achievable in others when multidisciplinary therapy and financial coverage of medical care were made available. 相似文献
17.
Treatment of recurrent focal segmental glomerulosclerosis post‐kidney transplantation in Australian and New Zealand children: A retrospective cohort study
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Disease recurrence affects around a third of renal transplants for children with FSGS and is associated with poor graft outcomes. Unfortunately, there are no large trials guiding treatment for recurrent FSGS. We aimed to describe current therapies and treatment response for recurrent FSGS in 4 centres in Australia and New Zealand. Data were collected on children (age <18 years) with recurrent FSGS (1990‐2015). We reviewed patient charts to obtain clinical information. Ethics approval was obtained from the relevant boards. Complete records were available on 24 patients (62% female, 54% Caucasian). Median time to first recurrence was 4 days (IQR 2‐5 days). There were 14 separate treatment regimens, involving an average of 2 agents. The most common therapies were plasma exchange (20/24 patients, 83%), cyclosporin (15/24, 63%), and methylprednisolone (9/24, 38%). Full remission was achieved in 15 (63%), partial remission in 2 (8%), and no remission in 7 (29%) patients. Of the patients with no remission, 5 lost their graft to recurrent disease and 1 to concurrent acute vascular rejection. The plethora of different treatment regimens reflects the poor evidence guiding management for recurrent FSGS. More research is needed to improve outcomes. 相似文献
18.
Nothing but NET: A review of norepinephrine transporter expression and efficacy of 131I‐mIBG therapy
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Keri A. Streby Nilay Shah Mark A. Ranalli Anne Kunkler Timothy P. Cripe 《Pediatric blood & cancer》2015,62(1):5-11
19.
Prognostic Factors for Outcome in Localized Extremity Rhabdomyosarcoma. Pooled Analysis from Four International Cooperative Groups
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Odile Oberlin MD Annie Rey Kenneth L.B. Brown MD Gianni Bisogno MD PhD Ewa Koscielniak MD PhD Michael C.G. Stevens MD Douglas S. Hawkins MD William H. Meyer MD PhD Trang H. La MD Modesto Carli MD James R. Anderson PhD 《Pediatric blood & cancer》2015,62(12):2125-2131
Background
Extremity rhabdomyosarcomas do not always show satisfactory outcomes. We analyzed data from 643 patients treated in 14 studies conducted by European and North American groups between 1983 and 2004 to identify factors predictive of outcome.Procedure
Clinical factors, including age; histology; site of primary (hand and foot vs. other); size; invasiveness (T stage); nodal involvement (N stage); and treatment factors, including post‐surgical group; chemotherapy type and duration; radiotherapy; and treatment (before or after 1995); were evaluated for impact on overall survival (OS).Results
5‐year OS were 67% (se 1.8). Multivariate analysis showed that lower OS correlated with age >3 years, T2 and N1 stage, incomplete initial surgery, treatment before 1995, and European cooperative group treatment. Patients with gross residual disease after initial incomplete resection/biopsy had similar outcomes in both continental groups. The better global survival of patients treated in American studies was accounted for by differences in outcome in the subset of those with grossly resected tumors (OS 86% [se 3] for COG patients vs. 68% [se 4] for European patients (P = 0.004)). When excluding chemotherapy duration from the model, analysis in this subset of patients showed that cooperative group (P = 0.001), site (P = 0.001), and T stage (P = 0.05) were all significant. However, after adding duration of chemotherapy (≥27 weeks) to the model, only primary site remained significant (P = 0.006).Conclusion
This meta‐analysis confirms the role of many established prognostic factors but identifies for the first time that chemotherapy duration may have an impact on outcome in patients with grossly resected tumors. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc. 相似文献20.
Salah Ali Sami AlThubaiti Samuele Renzi Joerg Krueger KY Chiang Ahmed Naqvi Tal Schechter Angela Punnett Muhammad Ali 《Pediatric transplantation》2019,23(1)
EBV‐related PTLD developing after HSCT is a potentially life‐threatening disease. HLH is uncommon after allogeneic HSCT. Data on outcome of patients with PTLD and concomitant HLH after allogeneic HSCT are limited. In this retrospective study, we collected demographic, clinical, laboratory, and outcome data for 408 patients who underwent allogeneic HSCT from 2006 to 2015. Graft source included CB (n = 135; 33.1%), PBSCs (n = 34; 8.3%), and BM (n = 239; 58.6%). Eight out of 408 patients (2%) developed EBV‐PTLD with a median age at HSCT of 5.9 years (range: 2.3‐17.3). All eight patients received ATG as part of the conditioning regimen. Graft source was PBSC in three patients (37.5%), BM in four patients (50%), and CB in one patient (12.5%). Donors were matched unrelated in five patients (62.5%) and matched sibling in three patients (37.5%). Seven out of eight patients developed EBV‐PTLD within the first 100‐day post‐HSCT. Lymph node biopsy revealed early lesions in three patients, polymorphic in three patients, and monomorphic PTLD in two patients. Three patients (37.5%) died within 1 month of EBV‐PTLD diagnosis. All deceased patients developed HLH manifestations with two of them meeting HLH diagnostic criteria and one having an incomplete workup. PTLD after allogeneic HSCT with manifestations of HLH is associated with high mortality. Early identification and treatment of EBV‐PTLD seems imperative to control the disease, especially if signs of HLH are evolving. 相似文献