scholarly journals Predictors of Relapse Risk Do Not Influence Time to "Cure" in Children and Young Adults with Acute Lymphoblastic Leukaemia

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 2306-2306
Author(s):  
Sujith Samarasinghe ◽  
Ajay Vora ◽  
Nicholas John Goulden ◽  
Grace Antony ◽  
Anthony V. Moorman
Keyword(s):  
Young Adults ◽  
Acute Lymphoblastic Leukaemia ◽  
Excess Mortality ◽  
Survival Rates ◽  
Relative Survival ◽  
Relapse Risk ◽  
Children And Young Adults ◽  
Good Risk ◽  
Risk Of Relapse

Abstract After how many years from diagnosis/completing treatment should children and young adults with acute lymphoblastic leukaemia be considered "cured"? Clinical trials generally report 5 year event free survival because the risk of relapse after that time-point is very low and this is assumed to mean cure. Indeed, the risk of relapse after four years from completing therapy was <1% for patients treated on St Jude's trial Total Therapy XV and they proposed that length of follow-up should be considered to be the "time to cure". The group had previously shown that excess deaths beyond ten years in non-irradiated patients was similar to the general population whereas irradiated patients had a greater risk of excess mortality due to the development of secondary neoplasms. However, there was limited information about the time to cure and relative survival in sub-groups with variable relapse risk. We examined the long-term outcome of patients treated on an MRD stratified protocol UKALL 2003 that recruited patients between 2003-2011 to determine time to cure for the overall trial population and prognostic sub-groups defined by age, sex, immunophenotype, cytogenetics and MRD response. There were a total of 3113 patients eligible for analysis with a median follow up of 9.4 years (only 20 patients received cranial irradiation). Relative survival rates were estimated using nationwide population mortality rates as the baseline accounting for age, sex and calendar year (Office of National Statistics 2018 release). The relative survival of UKALL2003 patients compared to equivalent children in the UK population was 96% (95% CI 95.5-96.7). Relative survival did not differ by sex but was significantly worse for patients >10 years compared with younger patients (p<0.001). As expected, patients with good risk cytogenetics ( ETV6-RUNX1, and high hyperdiploidy) and negative bone marrow Ig/TCR MRD at end of induction (EOI) had better relative survival rates and lower excess mortality (all p<0.001) compared with patients who had adverse risk factors (high risk genetics and EOI MRD+). To estimate the time to a cure, we calculated the conditional probability of relapse at 12 years for patients who had survived event to the start of each follow-up year. Although the initial risk of relapse differ significantly by sex, age, MRD and genetics the risk of relapse for all subgroups quickly coalesced at around 5-6 years (Figure 1). Furthermore, the time to cure, defined as a relapse risk <1% (dashed blue line of figure), was similar across all the subgroups at 6-7 years. In conclusion, the relative survival of young patients with good risk cytogenetics and EOI MRD negative status approaches that of their normal peers. However, regardless of prognosis, the time to cure is similar across risk groups. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.

Clinical Relevance of Conditional Survival of Cancer Patients in Europe: Age-Specific Analyses of 13 Cancers

2010 ◽  
Vol 28 (15) ◽  
pp. 2520-2528 ◽  
Author(s):  
Maryska L.G. Janssen-Heijnen ◽  
Adam Gondos ◽  
Freddie Bray ◽  
Timo Hakulinen ◽  
David H. Brewster ◽  
...  
Keyword(s):  
Cancer Survivors ◽  
Excess Mortality ◽  
Age Groups ◽  
Relative Survival ◽  
Cancer Registries ◽  
Initial Diagnosis ◽  
Accurate Information ◽  
Testis Cancer ◽  
Corpus Uteri

Purpose When cancer survivors wish to receive accurate information on their current prognosis during follow-up, conditional 5-year relative survival may be most suitable. We have estimated conditional 5-year relative survival for 13 cancers using a large European database—European Network for Indicators on Cancer (EUNICE)—of 10 dedicated long-standing cancer registries across Europe. Patients and Methods Patients age 15 years and older diagnosed between 1985 and 2004 were included. Conditional 5-year relative survival for each age group was computed for every additional year survived up to 10 years. Period analysis with follow-up period 2000 to 2004 was used. Results All patients with cutaneous melanoma or colorectal, endometrial, or testis cancer and younger patients with stomach, glottis, cervix, ovary, or thyroid cancer or non-Hodgkin's lymphoma exhibited hardly any excess mortality (conditional 5-year relative survival > 95%) given that they were alive at a defined time point within 10 years of initial diagnosis. However, patients with supraglottis, lung, breast, and kidney cancer, as well as older patients with most cancers exhibited substantial excess mortality (conditional 5-year relative survival < 90%). Initial differences in relative survival at diagnosis between age groups largely disappeared with time since initial diagnosis for melanoma, or stomach, colorectal, corpus uteri, or testicular cancer but persisted for patients diagnosed with other tumors. Differences between stage groups became smaller over time or disappeared. Conclusion Conditional relative survival shows clinically relevant variations according to time since diagnosis, type of cancer, and age, and can help serve as a guide for cancer survivors in planning for their future and for doctors in planning schedules for surveillance.

Pacemaker use for the treatment of reflex-mediated syncope: 40-year experience at a single paediatric institution

2021 ◽  
pp. 1-6
Author(s):  
Thomas Huang ◽  
Edward O’Leary ◽  
Mark E. Alexander ◽  
Laura Bevilacqua ◽  
Francis Fynn-Thompson ◽  
...  
Keyword(s):  
Young Adults ◽  
Atrioventricular Block ◽  
Sinus Bradycardia ◽  
Pacemaker Implantation ◽  
Complication Rates ◽  
Single Centre Experience ◽  
Procedural Complications ◽  
Children And Young Adults ◽  
Dual Chamber Pacemakers

Abstract Introduction: Reflex-mediated syncope occurs in 15% of children and young adults. In rare instances, pacemakers are required to treat syncopal episodes associated with transient sinus pauses or atrioventricular block. This study describes a single centre experience in the use of permanent pacemakers to treat syncope in children and young adults. Materials and methods: Patients with significant pre-syncope or syncope and pacemaker implantation from 1978 to 2018 were reviewed. Data collected included the age of presentation, method of diagnosis, underlying rhythm disturbance, age at implant, type of pacemaker implanted, procedural complications and subsequent symptoms. Results: Fifty patients were identified. Median age at time of the first syncopal episode was 10.2 (range 0.3–20.4) years, with a median implant age of 14.9 (0.9–34.3) years. Significant sinus bradycardia/pauses were the predominant reason for pacemaker implant (54%), followed by high-grade atrioventricular block (30%). Four (8%) patients had both sinus pauses and atrioventricular block documented. The majority of patients had dual-chamber pacemakers implanted (58%), followed by ventricular pacemakers (38%). Median follow-up was 6.7 (0.4–33.0) years. Post-implant, 4 (8%) patients continued to have syncope, 7 (14%) had complete resolution of their symptoms, and the remaining reported a decrease in their pre-syncopal episodes and no further syncope. Twelve (24%) patients had complications, including two infections and eight lead malfunctions. Conclusions: Paediatric patients with reflex-mediated syncope can be treated with pacing. Complication rates are high (24%); as such, permanent pacemakers should be reserved only for those in whom asystole from sinus pauses or atrioventricular block has been well documented.

Long-term follow-up of children and young adults with autoimmune hepatitis treated with cyclosporine

2019 ◽  
Vol 51 (5) ◽  
pp. 712-718 ◽  
Author(s):  
Silvia Nastasio ◽  
Marco Sciveres ◽  
Lorenza Matarazzo ◽  
Cristina Malaventura ◽  
Francesco Cirillo ◽  
...  
Keyword(s):  
Young Adults ◽  
Autoimmune Hepatitis ◽  
Long Term Follow Up ◽  
Children And Young Adults

scholarly journals Endocrine Deficiency As a Function of Radiation Dose to the Hypothalamus and Pituitary in Pediatric and Young Adult Patients With Brain Tumors

2018 ◽  
Vol 36 (28) ◽  
pp. 2854-2862 ◽  
Author(s):  
Ralph E. Vatner ◽  
Andrzej Niemierko ◽  
Madhusmita Misra ◽  
Elizabeth A. Weyman ◽  
Claire P. Goebel ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Young Adults ◽  
Young Adult ◽  
Thyroid Hormone ◽  
Radiation Dose ◽  
Brain Tumors ◽  
Adrenocorticotropic Hormone ◽  
Hormone Deficiency ◽  
Children And Young Adults

Purpose There are sparse data defining the dose response of radiation therapy (RT) to the hypothalamus and pituitary in pediatric and young adult patients with brain tumors. We examined the correlation between RT dose to these structures and development of endocrine dysfunction in this population. Materials and Methods Dosimetric and clinical data were collected from children and young adults (< 26 years of age) with brain tumors treated with proton RT on three prospective studies (2003 to 2016). Deficiencies of growth hormone (GH), thyroid hormone, adrenocorticotropic hormone, and gonadotropins were determined clinically and serologically. Incidence of deficiency was estimated using the Kaplan-Meier method. Multivariate models were constructed accounting for radiation dose and age. Results Of 222 patients in the study, 189 were evaluable by actuarial analysis, with a median follow-up of 4.4 years (range, 0.1 to 13.3 years), with 31 patients (14%) excluded from actuarial analysis for having baseline hormone deficiency and two patients (0.9%) because of lack of follow-up. One hundred thirty patients (68.8%) with medulloblastoma were treated with craniospinal irradiation (CSI) and boost; most of the remaining patients (n = 56) received involved field RT, most commonly for ependymoma (13.8%; n = 26) and low-grade glioma (7.4%; n = 14). The 4-year actuarial rate of any hormone deficiency, growth hormone, thyroid hormone, adrenocorticotropic hormone, and gonadotropin deficiencies were 48.8%, 37.4%, 20.5%, 6.9%, and 4.1%, respectively. Age at start of RT, time interval since treatment, and median dose to the combined hypothalamus and pituitary were correlated with increased incidence of deficiency. Conclusion Median hypothalamic and pituitary radiation dose, younger age, and longer follow-up time were associated with increased rates of endocrinopathy in children and young adults treated with radiotherapy for brain tumors.

The Ozaki Procedure With CardioCel Patch for Children and Young Adults With Aortic Valve Disease: Preliminary Experience – a Word of Caution

2019 ◽  
Vol 10 (6) ◽  
pp. 724-730 ◽  
Author(s):  
Sian C. Chivers ◽  
Carine Pavy ◽  
Ricky Vaja ◽  
Cesare Quarto ◽  
Olivier Ghez ◽  
...  
Keyword(s):  
Young Adults ◽  
Aortic Valve ◽  
Adult Population ◽  
Published Data ◽  
Bovine Pericardial Patch ◽  
Valve Position ◽  
Children And Young Adults ◽  
Ischemic Attack

Background: The Ozaki procedure is a surgical technique for patients with significant aortic stenosis or regurgitation or both where valve repair cannot be performed. Individual cusps are cut from glutaraldehyde-treated autologous pericardium or bovine pericardium and implanted into the aortic valve position. Encouraging results have been reported within the adult population. There are limited published data on success of this procedure in younger patients. Methods: We present a series of five children and young adults who underwent the Ozaki procedure with neoaortic valve cusps made from CardioCel, a decellularized bovine pericardial patch treated with a monomeric glutaraldehyde. Results: There were no complications in the initial postoperative period and short inpatient stay. At a mean follow-up of 29.6 months (range: 22-36 months), 4 patients had no evidence of stenosis and 3 patients had trivial or no regurgitation from the neoaortic valve. Overall, two patients had complications related to the valve and underwent reintervention during the follow-up period with a Ross procedure. One of these patients who was not taking long-term anticoagulation experienced a transient ischemic attack. Conclusions: Our experience demonstrates that the Ozaki procedure with CardioCel in pediatric and young adult patients should be approached with caution. Further research with larger groups of pediatric patients, comparison of different graft materials, and longer follow-up is required to ascertain long-term success in children.

scholarly journals Deep brain stimulation in children and young adults with secondary dystonia: the Children's Hospital Los Angeles experience

2013 ◽  
Vol 35 (5) ◽  
pp. E7 ◽  
Author(s):  
Joffre E. Olaya ◽  
Eisha Christian ◽  
Diana Ferman ◽  
Quyen Luc ◽  
Mark D. Krieger ◽  
...  
Keyword(s):  
Young Adults ◽  
Deep Brain Stimulation ◽  
Brain Stimulation ◽  
Secondary Dystonia ◽  
Benefit Evaluation ◽  
Generalized Dystonia ◽  
Children And Young Adults ◽  
The Mean ◽  
Deep Brain

Background Dystonia is a movement disorder in which involuntary sustained or intermittent muscle contractions cause twisting and repetitive movements, abnormal postures, or both. It can be classified as primary or secondary. There is no cure for dystonia and the goal of treatment is to provide a better quality of life for the patient. Surgical intervention is considered for patients in whom an adequate trial of medical treatment has failed. Deep brain stimulation (DBS), specifically of the globus pallidus interna (GPi), has been shown to be extremely effective in primary generalized dystonia. There is much less evidence for the use of DBS in patients with secondary dystonia. However, given the large number of patients with secondary dystonia, the significant burden on the patients and their families, and the potential for DBS to improve their functional status and comfort level, it is important to continue to investigate the use of DBS in the realm of secondary dystonia. Object The objective of this study is to review a series of cases involving patients with secondary dystonia who have been treated with pallidal DBS. Methods A retrospective review of 9 patients with secondary dystonia who received treatment with DBS between February 2011 and February 2013 was performed. Preoperative and postoperative videos were scored using the Barry-Albright Dystonia Scale (BADS) and Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) by a neurologist specializing in movement disorders. In addition, the patients' families completed a subjective questionnaire to assess the perceived benefit of DBS. Results The average age at DBS unit implantation was 15.1 years (range 6–20 years). The average time to follow-up for the BADS evaluation from battery implantation was 3.8 months (median 3 months). The average time to follow-up for the subjective benefit evaluation was 10.6 months (median 9.5 months). The mean BADS scores improved by 9% from 26.5 to 24 (p = 0.04), and the mean BFMDRS scores improved by 9.3% (p = 0.055). Of note, even in patients with minimal functional improvement, there seemed to be decreased contractures and spasms leading to improved comfort. There were no complications such as infections or hematoma in this case series. In the subjective benefit evaluation, 3 patients' families reported “good” benefit, 4 reported “minimal” benefit, and 1 reported no benefit. Conclusions These early results of GPi stimulation in a series of 9 patients suggest that DBS is useful in the treatment of secondary generalized dystonia in children and young adults. Objective improvements in BADS and BFMDRS scores are demonstrated in some patients with generalized secondary dystonia but not in others. Larger follow-up studies of DBS for secondary dystonia, focusing on patient age, history, etiology, and patterns of dystonia, are needed to learn which patients will respond best to DBS.

Sign in / Sign up

Export Citation Format

Share Document