Patient journey and quality of life (QOL) among diffuse-type TGCT in the U.S.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e23565-e23565
Author(s):  
Nicholas M. Bernthal ◽  
Geert Spierenburg ◽  
Michiel van de Sande ◽  
John H. Healey ◽  
Silvia Stacchiotti ◽  
...  

e23565 Background: Diffuse Tenosynovial Giant Cell Tumor (d-TGCT) is a rare, locally aggressive neoplasm of joint and tendon sheath synovia, which may severely affect patients (pts) QOL due to pain, limited joint function and destruction of bone and local tissues. This study analyzes real-world data to describe d-TGCT pt journey and QOL in the US. Methods: The TGCT Observational Platform Project (TOPP), the first non-interventional prospective disease registry, enrolled d-TGCT pts at 10 Western European (EU) and 2 US sarcoma centers from Nov 2016 to Mar 2019, with prospective follow-up of 24 mo. Pts demographics, disease history and ongoing medical treatment and pt-reported outcomes (PROs) (pain, stiffness, PROMIS Physical Function [PF] and EQ-5D) were collected at baseline and every 6 mo during follow-up periods. Descriptive analyses from baseline compared US pts with Western EU pts (Table). Results: Of 166 d-TGCT pts, 30 were enrolled in the US. Mean age was 40.6 ± 12.82 y, with 63.3% female and 66.7% with tumor in knee. Median time from first symptom to diagnosis was 12 mo (range; 0–153). 28 pts (93.3%) had surgery, 4 pts (13.3%) had tyrosine kinase inhibitors (TKI), no pts had radiotherapy. 18 pts (60%) experienced >1 locoregional recurrence. Median Pain Severity Score (PSS) and Pain Interference Score (PIS) were 2.8 and 3.0, respectively. Median PROMIS PF Score was 39.8. Mean EQ-5D index score and visual analogue scale (VAS) were 0.73 ±0.229 and 75.5 ±13.31, respectively. Compared with Western EU pts, US pts reported similar PROs and QOL, were younger, had shorter delay in diagnosis, and lower use of systemic therapy or radiotherapy (Table). Conclusions: d-TGCT affects a relatively young population in the US, similar to Western EU, resulting in serious impairment in daily activities and QOL. Shorter diagnostic delay and less frequent use of non–surgical approach was observed in the US. [Table: see text]

Author(s):  
Abderrahmane Jallouli ◽  
Mariama Jarti ◽  
Marj Zohour Haida ◽  
Mouna El Bouatmani ◽  
Adil Ait Errami ◽  
...  

Rectal gastrointestinal stromal tumors (GIST) are extremely rare, accounting for approximately 0.1% of all rectal tumors. Diagnosis is based on histological and immunohistochemical confirmation. We report the case of a 38-year-old patient with a rectal GIST revealed by chronic rectal bleeding associated with rectal tenesmus, abdominal pain with painful contractions and frequent evacuations evolving in a context of altered general state. Rectoscopy was used to visualize the tumor mass and to take biopsies; their histological and immunohistochemical study revealed a rectal location of a gastrointestinal stromal tumor. The extension assessment was negative. After discussion of the case in a multidisciplinary consultation meeting, management consisted of initially putting the patient on Imatinib given the large tumor size, the degree of local invasion and the location of the mass (lower rectum), with regular follow-up in order to schedule a less invasive surgical resection later. Despite the rarity of rectal GIST, early diagnosis is necessary to avoid progression to locoregional invasion complicating some surgical resections given the anatomical constraints of the pelvic region. Hence the interest of neoadjuvant therapy with tyrosine kinase inhibitors allowing in some cases a decrease in tumor volume, a regression of the degree of local invasion and a decrease in the morbidity of the surgery in order to improve the patient's quality of life.


2020 ◽  
Vol 29 (Suppl 3) ◽  
pp. s191-s202 ◽  
Author(s):  
Karin A Kasza ◽  
Kathryn C Edwards ◽  
Zhiqun Tang ◽  
Cassandra A Stanton ◽  
Eva Sharma ◽  
...  

ObjectiveTo report on demographic and tobacco product use correlates of tobacco product initiation (cigarettes, electronic nicotine delivery systems (ENDS), cigars, hookah and smokeless tobacco) among the US population.DesignData were from the first three waves (2013–2016) of the Population Assessment of Tobacco and Health Study, a nationally representative, longitudinal cohort study of US youth (aged 12–17 years) and adults (aged 18+ years). Never users of at least one type of tobacco product at Wave 1 (W1, 2013/14) or Wave 2 (W2, 2014/15) were included (n=12 987 youth; n=25 116 adults). Generalised estimating equations were used to evaluate the association between demographic and tobacco product use characteristics at baseline, and tobacco product initiation at follow-up (ever, past 30 day (P30D), frequent (use on 20 or more of thepast 30 days)) over two 1-year periods (W1–W2 and W2–Wave 3).ResultsYouth aged 15–17 years were more likely than youth aged 12–14 years and adults aged 18–24 years were more likely than older adults to initiate P30D tobacco use across products; non-heterosexuals were more likely than heterosexuals to initiate P30D cigarette and ENDS use. Older adults were more likely than young adults, and males were more likely than females, to be frequent users of ENDS on initiation. Ever use of another tobacco product predicted P30D initiation of each tobacco product.DiscussionOther tobacco product use and age predict P30D tobacco initiation across products whereas associations with other demographic characteristics vary by product. Continued contemporary evaluation of initiation rates within the changing tobacco product marketplace is important.


Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4762-4762
Author(s):  
Herve Ghesquieres ◽  
Cedric Rossi ◽  
Fanny Cherblanc ◽  
Sandra Le Guyader ◽  
Fontanet Bijou ◽  
...  

Introduction: Lymphoma incidence continues to rise in France since early 80', although differently among subtypes. Recent improvements in patient survival in major lymphoma subtypes at populational level raise new questions about patient outcome (in specific subgroups) and survivorship (i.e. quality of life, long term sequelae). Numerous epidemiological studies have investigated factors related to lymphoma risk, but far fewer have addressed the extent to which socioeconomic status, social institutional context (i.e. healthcare system), social relationships, environmental context (exposures), individual behaviours (lifestyle) or genetic determinants influence lymphoma outcomes, especially in the general population. Moreover, the knowledge of the disease behaviour achieved from clinical trials data is partly biased because of patient selection. Study Design and Methods: The REALYSA ("REal world dAta in LYmphoma and Survival in Adults") study is a real-life multicentric cohort set in France areas mostly covered by population-based cancer registries to study the prognostic value of epidemiological, clinical and biological factors with a prospective 9-year follow up. ClinicalTrials.gov identifier is NCT03869619. We aim to include 6000 patients over 4 years. Adult patients without lymphoma history and newly diagnosed of one of the following 7 lymphoma subtypes (diffuse large B cell (DLBCL), follicular (FL), marginal zone (MZL), mantle cell (MCL), Burkitt, Hodgkin (HL), T-cell (T-NHL)) are offered to participate during a medical consultation with their hematologist. Exclusion criteria are: having already received anti-lymphoma treatment (except pre-phase) and having a documented HIV infection. After having given signed informed consent, participants fill in three auto-questionnaires regarding lifelong history of residences and occupations, quality of life (QoL / QLQ-C30 questionnaire) and social support (SS / SSQ6 questionnaire). Patients are then interviewed to collect their sociodemographic characteristics, medical and familial history, professional and domestic exposures to major chemicals and pesticides, lifestyle and women health. Clinical data are obtained using patients medical records, including care pathway, medical history, concomitant treatments, initial diagnosis characteristics, nodal/extra-nodal involvement, exams performed, staging, laboratory data, serologic tests, geriatric screening (G8 questionnaire), treatments received (including pre-phase, detailed treatment phases and molecules, reasons for treatment discontinuation), progressions, and treatment response evaluation. Biological samples at baseline and during treatment are collected including plasma and peripheral mononuclear cells. Additionally, a virtual tumor biobank is constituted for baseline tumor samples. The diagnosis will be ensured thanks to the review of French Lymphopath network. Follow-up, including clinical outcomes, new morbidities, lifestyle, professional situation, QoL, SS, fertility, health behavior, are collected every 6 months in the first 3 years and every year thereafter. Results: A pilot phase was implemented between November 2018 and June 2019 in 7 French hospitals/clinics. By June 30, 328 patients were recruited. Biological samples at baseline were obtained for 81% of included patients (n=265). 52% were male and 48% were female. The median age was 62 years (range: 18-95). The histological subtypes were the following (n=308 patients with complete data): 132 DLBCL (42.8%); 59 FL (19.5%); 52 HL (16.9%); 29 MCL (9.4%); 22 MZL (7.1%); 13 T-NHL (4.2%); 1 other (0.3%). We observed a good adherence to clinical research process despite the complexity of data collection. An extension phase with 10 additional centres will be launched during the last 2019 trimester. Discussion: The pilot phase of REALYSA study showed a good compliance to study guidelines and a good quality of data collected at baseline. Consequently, the study design is prospectively feasible in real-life setting. This cohort will constitute an innovative platform for clinical, biological, epidemiological and socio-economical research projects. Disclosures Oberic: Takeda: Membership on an entity's Board of Directors or advisory committees; Janssen: Honoraria; Roche: Membership on an entity's Board of Directors or advisory committees. Salles:Amgen: Honoraria, Other: Educational events; Novartis, Servier, AbbVie, Karyopharm, Kite, MorphoSys: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Educational events; BMS: Honoraria; Roche, Janssen, Gilead, Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Educational events; Takeda: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Educational events; Merck: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Autolus: Consultancy, Membership on an entity's Board of Directors or advisory committees; Epizyme: Consultancy, Honoraria.


Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4758-4758
Author(s):  
Javier Pinilla-Ibarz ◽  
Maryam Navaie ◽  
Jessica C. Smith ◽  
Anne C. Beaubrun ◽  
Lauren E. McCullough ◽  
...  

Abstract Abstract 4758 Introduction: Among patients with chronic myeloid leukemia (CML), the success of clinical care has traditionally been evaluated in terms of survival, defined clinical events (e.g., transfusions, hospital days), and test results (e.g., major molecular or cytogenetic response). However, some of these measures may not adequately describe whether patients actually show improvements in health-related quality of life (HRQoL). Conceptually, increases in HRQoL are derived from two possible effects of pharmacotherapies: (a) comparative improvement of disease symptoms, and (b) reduction of therapy-related adverse events (AEs). With the advent of tyrosine kinase inhibitors (TKIs), the leading agents in CML treatment, improved morbidity and delayed mortality have been achieved with astonishing success. Moreover, through rigorously conducted research, much has been learned about the tolerability profiles to these agents with careful delineation of AEs. Recently, there has been a growing recognition by the clinical community about the need to examine the impact of TKI-related AEs on HRQoL from the patient's perspective as well as the perspectives of their health care proxies including physicians and family caregivers. As summarized in Figure 1, the triangulation of these three perspectives in cancer care has been shown to provide valuable information that informs clinical decision-making, improves adherence to prescribed treatment regimens, and optimizes care management. Thus, the purpose of this study was to evaluate the CML literature to determine the extent to which the consequences of TKI-related AEs on HRQoL has been explored from the comparative perspectives of physicians, patients and family caregivers. Methods: A systematic literature review of PubMed conducted by three independent reviewers identified 44 articles using combinations of the following broad key words: CML, HRQoL, imatinib, nilotinib, dasatinib, tyrosine kinase inhibitors, adverse events, side effects and toxicity. Articles that were economic focused (n=11), commentaries (n=9), systematic reviews without HRQoL as an endpoint measure (n=7), non-English publications (n=4), studies on pediatric patients or those without CML as a primary diagnosis (n=5), non-TKI specific (n=4) or multiple publications on the same patient population (n=1) were excluded from review. The remaining 3 articles employed different study designs including cross-sectional, longitudinal and randomized clinical trial approaches with HRQoL as a primary endpoint. Collectively, they involved 1,587 CML patients treated with TKIs. Results: The studies were limited to patients in the chronic phase of CML when HRQoL was likely to be at its highest. The average follow-up time was short, ranging between 6 to 18 months with no long-term follow-up. None of the studies provided the comparative perspective of the physician or the primary family caregiver in relation to the patient with regards to either short-term or long-term impact of AEs on HRQoL. Rather, the studies were centered solely on the patient's reported HRQoL secondary to their experience with TKI therapy but none actually linked the type, frequency or severity of AEs to the various dimensions of HRQoL. Therefore, no information was available on which AEs have the greatest effect on what dimension of HRQoL. The most commonly reported AEs that adversely impacted HRQoL included (a) non-hematologic events related to gastroenterology, dermatology, musculoskeletal system and other (e.g., edema); and (b) hematologic events such as neutropenia, thrombocytopenia, and anemia. Across the studies, the most consistently distressing AE experienced by >80% of patients was “lack of energy”. Desired life activities, such as being able to return to work as well as the ability to participate in social and family engagements, were also rated highly in relation to better HRQoL. Conclusions: On the whole, little to no data are available on the temporal impact of TKI-related AEs on the multiple dimensions of HRQoL. This substantial dearth of information in the CML literature is a stark contrast to other types of cancers and therapeutic agents where the myriad of factors that affect the patient's HRQoL have been investigated from the perspectives of key stakeholders in the health care paradigm, resulting in optimal clinical decision-making and improved adherence to prescribed regimens. Disclosures: Pinilla-Ibarz: Novartis Pharmaceuticals Corporation: Consultancy. Navaie:Novartis Pharmaceuticals Corporation: Consultancy. Smith:Novartis Pharmaceuticals Corporation: Consultancy. Beaubrun:Novartis Pharmaceuticals Corporation: Consultancy. McCullough:Novartis Pharmaceuticals Corporation: Consultancy. Hickey:Novartis Pharmaceuticals Corporation: Consultancy. Toseland:Novartis Pharmaceuticals Corporation: Consultancy.


2021 ◽  
Vol 5 (3) ◽  
Author(s):  
Aurora Parodi ◽  
Valeria Maria Messina ◽  
Manuela Martolini ◽  
Shpresa Haxhiaj ◽  
Emanuele Claudio Cozzani

Le lesioni da decubito, le ulcere vascolari e il piede diabetico rappresentano le lesioni cutanee croniche maggiormente diffuse in età geriatrica. La lesione cutanea cronica presenta un elevato rischio infettivo ed un management complesso che richiede terapie mirate ed un iter di trattamento specifico. Il metodo TIME (Tissue management control of Infection and inflammation Moisture imbalance advancement of the Epithelial edge of the wound), costituisce il gold standard per il trattamento delle lesioni cutanee croniche poiché consente di controllare l’infezione e il grado di macerazione della ferita anche nei margini epiteliali. La soluzione di ipoclorito di sodio alla concentrazione dello 0,05%, oggetto di numerosi studi in letteratura, grazie ad un ampio spettro germicida ed alla sua elevata compatibilità tissutale rappresenta il metodo di disinfezione d’elezione per il trattamento delle lesioni cutanee croniche. La qualità di vita (Quality of Life, QoL) di un paziente affetto da lesione cutanea cronica può essere fortemente compromessa. La formazione di un team sanitario multidisciplinare per la gestione del patient journey può favorire il percorso di guarigione, facilitare la gestione della lesione nella quotidianità e migliorare la QoL del paziente. La telemedicina spicca tra le modalità innovative di gestione del wound care sperimentate da un’equipe di specialisti del territorio ligure negli ultimi mesi a seguito della pandemia COVID-19. La pratica della telemedicina si è rivelata particolarmente utile nel follow up della lesione cronica a fronte di un adeguato impiego di strumenti tecnologici che permettano un’elevata qualità di immagini.


2021 ◽  
Vol 8 (Supplement_1) ◽  
pp. S765-S766
Author(s):  
Madison T Preib ◽  
Fanny S Mitrani-Gold ◽  
Ziyu Lan ◽  
Xiaoxi Sun ◽  
Ashish V Joshi

Abstract Background Gonorrhea (GC) is a major public health threat in the US. The Centers for Disease Control and Prevention (CDC) estimated direct healthcare costs of &271 million in 2018. CDC 2015 guidelines (applicable up to December 18, 2020) recommended cephalosporin plus azithromycin for GC. We used real-world data to assess patterns of inappropriate or suboptimal (IA/SO) or appropriate and optimal (AP&OP) antibiotic (AB) prescription (by CDC 2015 guidelines), and related healthcare costs, in US patients with uncomplicated urogenital GC (uUGG) diagnosed from July 1, 2013–June 30, 2018. Methods A retrospective cohort study of IBM MarketScan data (commercial/Medicare claims) in patients ≥ 12 years old with uUGG. Eligible patients had an AB prescription ±5 days of uUGG diagnosis (index date) and continuous health-plan enrollment with ≥ 6 months’ baseline/≥ 12 months’ follow-up data. Patients with complicated urogenital GC were excluded. Patients were stratified by AB prescription (IA/SO or AP&OP; defined in Table 1) during the first uUGG episode (ie, within 30 days of index). Generalized linear models were used for multivariate analysis. Table 1. Definitions of appropriateness of AB prescriptions Results Of 2847 patients with uUGG (58.5% male), 77.1% had an IA/SO prescription (mostly due to IA AB class [~82.0%] and duration [24.0%]), while only 22.9% had an AP&OP prescription; uUGG episodes were more frequent with IA/SO (n=2386) than AP&OP (n=714) prescriptions during follow-up. Patients with IA/SO prescriptions had higher GC-related total adjusted costs per patient (PP) per index episode (&196) vs those with AP&OP prescriptions (&124, p < 0.0001; Figure). Patients with IA/SO prescriptions also had higher GCrelated total adjusted costs PP during follow-up (&220) vs those with AP&OP prescriptions (&148, p < 0.0001), mostly driven by higher outpatient ambulatory and emergency room (ER) adjusted costs with IA/SO (&148 and &71, respectively) vs AP&OP prescriptions (&129 and &12, respectively, p ≤ 0.0152; Figure). ER visits PP at index and during follow-up were higher with IA/SO vs AP&OP prescriptions (p < 0.0001; Table 2). Figure. GC-related costs per patient with uUGG, stratified by appropriateness of AB prescription* Table 2. GC-related HRU per patient with uUGG, stratified by AB prescription Conclusion Most patients with uUGG were not prescribed treatments in accordance with CDC 2015 guidelines. High IA/SO AB prescriptions and associated healthcare costs suggest an unmet need for improved prescribing practices for uUGG in the US. Disclosures Madison T. Preib, MPH, STATinMED Research (Employee, Former employee of STATinMED Research, which received funding from GlaxoSmithKline plc. to conduct this study) Fanny S. Mitrani-Gold, MPH, GlaxoSmithKline plc. (Employee, Shareholder) Ziyu Lan, MSc, STATinMED Research (Employee, Employee of STATinMED Research, which received funding from GlaxoSmithKline plc. to conduct this study) Xiaoxi Sun, MA, STATinMED Research (Employee, Employee of STATinMED Research, which received funding from GlaxoSmithKline plc. to conduct this study) Ashish V. Joshi, PhD, GlaxoSmithKline plc. (Employee, Shareholder)


Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 230-230
Author(s):  
Shruti Chaturvedi ◽  
Hashim Abbas ◽  
Keith R. McCrae

Abstract Background and Objective: The advent of plasma exchange has led to a dramatic improvement in the survival of patients with thrombotic thrombocytopenic purpura (TTP). Recovery from TTP is usually complete, but may be complicated by a significant rate of relapse, particularly in the first year after the acute episode. Recent reports also suggest that survivors of TTP may have a poorer health related quality of life than previously estimated. We followed a cohort of 100 patients diagnosed with TTP to estimate the burden of chronic renal impairment, hypertension, diabetes, stroke, depression and other psychiatric and neurologic conditions in this group. Patients and Methods: A total of 100 consecutive patients treated for TTP at the Cleveland Clinic from January 2000 to March 2012 were identified. Nine patients died and the remaining 91 patients were followed until death or date of last clinical contact. The point prevalence of chronic kidney disease, hypertension, diabetes, stroke, major depression and other neurologic and psychiatric conditions was compared to expected values in the reference US population [based on National Health and Nutrition Examination Survey (NHANES) data] adjusted for age and sex. Results: Of the 91 patients who survived a first episode of TTP, 69 (75.8%) were female, with an age range of 16 to 79 years (median 44 years). Fifty one percent of patients were Caucasian, 45% African American and 2% Hispanic. Sixty three percent of cases occurred without predisposing conditions while 12.1% were associated with autoimmune disease (5 with SLE, 2 with rheumatoid arthritis, one with SLE and rheumatoid arthritis, and one each with Sjogren's syndrome, dermatomyositis and mixed connective tissue disorder), 8% with pregnancy or the postpartum state, 6.6% with cancer and 5.5% with solid organ transplant. The median follow up time was 59 months (range 1-117 months). During this period, 18 patients suffered relapses (11 patients had 1 relapse, 6 patients had 2 relapses, and 1 had three relapses). Compared to the reference US population, survivors of TTP had a higher prevalence of hypertension (61.5% versus 29.8%), chronic renal disease (26.4% versus 16.8%) and stroke (12.1% versus 1.87%). TTP survivors also had a high prevalence of neurologic disorders including migraines (n=9), multiple sclerosis (n=1), fibromyalgia (n=1) and restless leg syndrome (n=2). There was a five-fold higher incidence of major depression requiring pharmacologic treatment compared to the reference population (26.4% versus 5.4%). We also noted a significant prevalence of other mental health disorders such as anxiety disorders (n=15), bipolar disorder (n=2), adjustment disorder (n=1) and conversion disorder (n=1). The incidence of diabetes was lower than seen in the reference population (6.6% versus 14.4%). Conclusion: Survivors of TTP are at higher risk for poor health outcomes including chronic cardiovascular disease, neurologic disease and psychiatric conditions, all of which impair quality of life. These may be related to persistent organ damage from microvascular dysfunction during the acute episode of TTP, or from co-morbid conditions such as auto-immune diseases that are independent risk factors for poor outcomes. Our results highlight the importance of continued follow up in order to address the long-term health problems associated with TTP survivorship. Figure 1 Prevalence of health outcomes in TTP survivors compared to the US reference population. Figure 1. Prevalence of health outcomes in TTP survivors compared to the US reference population. Disclosures No relevant conflicts of interest to declare.


2020 ◽  
Vol 13 (Suppl_1) ◽  
Author(s):  
David Lin ◽  
Christine Majeski ◽  
Maral DerSarkissian ◽  
Matt Magestro ◽  
Cristi Cavanaugh ◽  
...  

Introduction: Real-world data describing acute pericarditis (AP) etiology in the US are limited. Data on the characteristics of recurrent pericarditis (RP) patients (pts) are also sparse. To fill this gap, our study assesses longitudinal data from a nationwide privately-insured population. Methods: OptumHealth Reporting and Insights employer claims data (1/2007-3/2017) were used. AP pts were identified and categorized as idiopathic or non-idiopathic etiology based on presence or absence of attributable conditions. Among idiopathic AP pts, a subgroup of RP pts was identified. Recurrence was defined as ≥2 AP events separated by >4 weeks. First recurrence date marked the index date. Pts aged ≥18 years with ≥12 months of continuous enrollment pre-index (baseline) were included. Results: Of 17,168 AP pts, 4,175 (24.3%) had non-idiopathic and 12,993 (75.7%) had idiopathic etiology (Table 1). Application of inclusion criteria left 8,822 idiopathic AP pts, of whom 1,604 (18.2%) had ≥1 recurrence during a mean observation period of 29 months. On average, idiopathic RP pts were aged 50.7 years, 51.6% female, and 42.3% had baseline history of hypertension, 23.8% of coronary artery disease, 11.7% of hypercholesterolemia, and 7.3% of myocardial infarction. Mean (±SD) time from initial AP diagnosis to first recurrence was 8.7 (±12.1) months and mean (±SD) number of recurrences was 1.7 (±1.3) per pt. In idiopathic RP pts with ≥4 years of follow-up after the initial AP diagnosis (N=512), 35.9% had ≥2, 18.2% had ≥3, and 9.8% had ≥4 recurrences within 4 years. Conclusions: The etiologic distribution and proportion of pts with RP are consistent with previous reports. About 36% of RP pts experience ≥2 recurrences after AP diagnosis over 4 years. RP represents a significant clinical burden for affected pts.


Hematology ◽  
2017 ◽  
Vol 2017 (1) ◽  
pp. 102-109 ◽  
Author(s):  
Francois-Xavier Mahon

Abstract Chronic myeloid leukemia (CML) is the best example of successful targeted therapy. Today, the overall survival of patients with CML treated by using tyrosine kinase inhibitors (TKIs) is very close to that of the healthy population. The current question is: how can we further ameliorate the clinical outcome of patients with CML? Clinical trials have shown that some patients with CML in the chronic phase who achieve sustained deep molecular responses on TKI therapy can safely suspend therapy with no evidence of relapse. The long follow-up studies and the number of eligible patients have now validated the concept of treatment-free remission (ie, the ability to maintain a molecular response after stopping therapy). It should be considered as the future criterion to evaluate the success of clinical trials, especially if we want to take into account the quality of life of patients in addition to the economic aspect. Because post-TKI discontinuation follow-ups have been increasing over time with no evidence of relapse in some patients, the next step for the coming decade will be to address the topic of CML cure.


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