Late outcomes in survivors of childhood acute myeloid leukemia: a report from the St. Jude Lifetime Cohort Study.

Cumulative burden of chronic health conditions and neurocognitive and physical function were examined among survivors of childhood acute myeloid leukemia (AML) treated with hematopoietic cell transplant (HCT; n = 66) or conventional therapy (CT; n = 67). Survivors and controls underwent a comprehensive clinical assessment, and health conditions were graded using a modified version of the Common Terminology Criteria for Adverse Events. By age 40 years, HCT and CT survivors had an average 17.4 (95% confidence interval [CI] 14.6-20.1) and 9.3 (7.7-11.1) grade 1-4 conditions versus 3.8 (3.3-4.2) in community controls. Compared to controls, HCT survivors had a higher prevalence of hypertriglyceridemia (45.5% vs. 18.3%), hypercholesterolemia (47.0% vs. 30.9%), hypothyroidism (27.3% vs. 4.0%), and primary hypogonadism (p < 0.001). CT survivors had a higher prevalence of cardiomyopathy (11.9% vs. 2.7%) and hypertension (53.7% vs. 44.3%). Neurocognitive impairment was elevated across all domains compared to controls but did not differ by treatment modality. Compared to controls, a higher proportion of HCT survivors had impairments in strength and endurance; whereas flexibility and mobility impairments were noted among CT survivors. Despite successful advances in childhood AML therapy, many therapeutic exposures remain unchanged. These findings support ongoing investigations of novel therapies and strategies to ameliorate the risk of late morbidities.

Long-Term Neurocognitive and Psychosocial Outcomes After Acute Myeloid Leukemia: A Childhood Cancer Survivor Study Report.

BACKGROUND: Survivors of childhood acute myeloid leukemia (AML) are vulnerable to medical late effects of treatment; however, less is known about their psychosocial outcomes. This study evaluated neurocognitive and psychosocial outcomes in long-term AML survivors treated with bone marrow transplantation (BMT) or intensive chemotherapy (IC) without BMT. METHODS: AML survivors (N = 482; median age at diagnosis = 8 [range = 0-20] years; median age at evaluation = 30 [range = 18-49] years) treated with BMT (n = 183) or IC (n = 299) and sibling controls (N = 3190; median age at evaluation = 32 [range = 18-58] years) from the Childhood Cancer Survivor Study were compared on emotional distress (Brief Symptom Inventory-18), neurocognitive problems (Childhood Cancer Survivor Study Neurocognitive Questionnaire), health-related quality of life (SF-36), and social attainment. Outcomes were dichotomized (impaired vs nonimpaired) using established criteria, and relative risks (RRs) were estimated with multivariable Poisson regression, adjusted for age at evaluation and sex. RESULTS: AML survivors were more likely than siblings to report impairment in overall emotional (RR = 2.19, 95% confidence interval [CI] = 1.51 to 3.18), neurocognitive (RR = 2.03, 95% CI = 1.47 to 2.79), and physical quality of life (RR = 2.71, 95% CI = 1.61 to 4.56) outcomes. Survivors were at increased risk for lower education (RR = 1.15, 95% CI = 1.03 to 1.30), unemployment (RR = 1.41, 95% CI = 1.16 to 1.71), lower income (RR = 1.39, 95% CI = 1.17 to 1.65), and not being married or having a partner (RR = 1.33, 95% CI = 1.17 to 1.51). BMT-treated survivors did not differ statistically significantly from IC-treated on any outcome measure. CONCLUSIONS: AML survivors are at increased risk for psychosocial impairment compared with siblings; however, BMT does not confer additional risk for psychosocial late effects compared with treatment without BMT.

Unmet supportive care needs and its relation to quality of life among adult acute leukaemia patients in China: a cross-sectional study.

BACKGROUND: Patients with acute leukaemia (AL) usually require prolonged periods of hospitalisation. The treatment and clinical symptoms may lead to patients' supportive care needs (SCNs) not being met and impairs their quality of life (QoL). Studies on QoL and SCNs among AL patients are limited. This study aimed to identify the unmet SCNs and its relation to QoL of adult AL patients in China. METHODS: This multicentre cross-sectional study recruited 346 participants to complete a self-developed questionnaire, detailing demographic information and disease-related variables. A 34-item Supportive Care Needs Survey (SCNS-SF34) was used to identify unmet SCNs, and the Functional Assessment of Cancer Therapy-Leukaemia (FACT-Leu) questionnaire measured patients' QoL. RESULTS: Unmet SCN rates for the 34 items ranged from17.6 to 81.7%. Patients' needs were high for health systems and information, but low in the sexual domain. The results reveal nine factors associated with the unmet SCNs of adult AL patients, including marital status, original residence, age, education, occupation, other diseases, chemotherapy course, disease course, and treatment stage (p < 0.05). The total score of the FACT-Leu negatively correlated with the SCNS-SF34 in the physical/daily living (r = - 0.527, p < 0.01), psychological (r = - 0.688, p < 0.01), sexual (r = - 0.170, p < 0.01), patient care and support (r = - 0.352, p < 0.01), and health systems and information (r = - 0.220, p < 0.01) domains. CONCLUSIONS: Adult AL patients exhibit a high demand for unmet SCNs, especially in the domain of health systems and information. There was a significant association between patients' unmet SCNs and QoL. Future research should develop tailored interventions to address the unmet SCNs of adult AL patients, to further improve their QoL.

Fear of disease progression and relevant correlates in acute leukemia patients prior to allogeneic hematopoietic stem cell transplantation.

OBJECTIVE: Prior to hematopoietic stem cell transplantation (HSCT), and despite the high objective risks associated with HSCT, fear of progression (FoP) has only sparsely been studied in patients with acute leukemia until now. The aim of this study was the assessment of the prevalence of FoP, and any relevant correlates and typical concerns. METHODS: We included 59 adult patients with acute leukemia who were tested prior to their first HSCT during an in-patient stay. Patients completed self-report measures assessing FoP (Fear of Progression Questionnaire-Short Form, FoP-Q-SF) and relevant correlates of FoP (eg, patients' physical state, depression, psychosocial distress, or social support). RESULTS: About one third of the patients (35.6%; n = 21) had high FoP. Higher FoP was associated with previous cancer diagnosis, as well as depression, anxiety, reduced physical functioning, lower mental health-related quality of life, and lower positive social support. The greatest fears reported by these patients reflected common fears of cancer patients (eg, fear about leaving their families behind), but also specific fears related to the current situation (eg, fear of severe medical treatments during the course of the illness). CONCLUSIONS: This was the first study providing an extensive analysis of FoP in leukemia patients prior to the extreme situation of HSCT. FoP is frequent and of major clinical importance for these patients. Hence, we recommend that patients undergo routine screening for FoP, to identify highly distressed patients at an early stage, and to offer targeted support.

Age at Diagnosis and Patient Preferences for Treatment Outcomes in AML: A Discrete Choice Experiment to Explore Meaningful Benefits.

BACKGROUND: The recent expansion of treatment options in acute myeloid leukemia (AML) has necessitated a greater understanding of patient preferences for treatment benefits, about which little is known. METHODS: We sought to quantify and assess heterogeneity of the preferences of AML patients for treatment outcomes. An AML-specific discrete choice experiment (DCE) was developed involving multiple stakeholders. Attributes included in the DCE were event-free survival (EFS), complete remission (CR), time in the hospital, short-term side effects, and long-term side effects. Continuously coded conditional, stratified, and latent-class logistic regressions were used to model preferences of 294 patients with AML. RESULTS: Most patients were white (89.4%) and in remission (95.0%). A 10% improvement in the chance of CR was the most meaningful offered benefit (P < 0.001). Patients were willing to trade up to 22 months of EFS or endure 8.7 months in the hospital or a two-step increase in long-term side effects to gain a 10% increase in chance of CR. Patients diagnosed at 60 years or older (21.6%) more strongly preferred to avoid short-term side effects (P = 0.03). Latent class analysis showed significant differences of preferences across gender and insurance status. CONCLUSIONS: In this national sample of mostly AML survivors, patients preferred treatments that maximized chance at remission; however, significant preference heterogeneity for outcomes was identified. Age and gender may affect patients' preferences. IMPACT: Survivor preferences for outcomes can inform patient-focused drug development and shared decision-making. Further studies are necessary to investigate the use of DCEs to guide treatment for individual patients.

Factors associated with quality of life of adult patients with acute leukemia and their family caregivers in China: a cross-sectional study.

BACKGROUND: Acute leukemia (AL) not only impairs the quality of life (QOL) of patients, but also affects that of their family caregivers (FCs). Studies on QOL of AL patients and their FCs are limited. This study aimed to evaluate the QOL of AL patients and their FCs, and to explore the factors associated with QOL of patients and of FCs. METHODS: A multicenter cross-sectional study was conducted. The QOL of 196 patient-FC dyads was assessed. The Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) was used for patients, and the 36-item Short-Form Health Survey (SF-36) was used for FCs. Independent-samples t-tests or one-way analysis of variance were used to compare QOL subscale scores between groups with different sociodemographic/clinical characteristics. Multiple regression analysis was conducted to identify the factors associated with QOL of AL patients and their FCs. RESULTS: The total FACT-Leu score for AL patients was 76.80 ± 16.44, and the physical component summary (PCS) and mental component summary (MCS) scores for FCs were 64.67 ± 15.44 and 52.50 ± 13.49, respectively. All QOL subscales for patients (t = 12.96-34.73, p < 0.001) and FCs (t = 2.55-14.36, p < 0.05), except role emotional (t = - 0.01, p = 0.993), were lower than those reported in previous studies. Sex, employment, and chemotherapy were significantly associated with total FACT-Leu score in AL patients (p < 0.05). Age, sex, marital status, education, employment, and relationship to patients were significantly associated with SF-36 PCS or MCS (p < 0.05). CONCLUSIONS: AL patients and their FCs both have lower QOL than the population in previous studies. These findings suggest that not only AL patients' physical and mental health but also overall family QOL should be assessed. Interventions supporting patient-FC dyads should be developed to improve their QOL.

Patient-Centered Insights on Treatment Decision Making and Living with Acute Myeloid Leukemia and Other Hematologic Cancers.

Expectations relating to treatment and survival, and factors influencing treatment decisions are not well understood in adult patients with acute myeloid leukemia. This study analyzed combined findings from a targeted literature review with patient-reported information shared on YouTube to further understand patient perspectives in hematologic cancers and, in particular, acute myeloid leukemia. The targeted literature review included articles concerning patient (aged ≥ 18 years) experiences or perspectives in acute myeloid leukemia or other hematologic cancers. YouTube video selection criteria included patients (aged ≥ 60 years) with self-reported acute myeloid leukemia. In total, 26 articles (13 acute myeloid leukemia-specific and 14 other hematologic cancers, with one relevant to both populations) and 28 videos pertaining to ten unique patients/caregivers were identified. Key concepts reported by patients included the perceived value of survival for achieving personal and/or life milestones, the emotional/psychological distress of their diagnosis, and the uncertainties about life expectancy/prognosis. Effective therapies that could potentially delay progression and extend life were of great importance to patients; however, these were considered in terms of quality-of-life impact and disruption to daily life. Many patients expressed concerns regarding the lack of treatment options, the possibility of side effects, and how their diagnosis and treatment would affect relationships, daily lives, and ability to complete certain tasks. Both data sources yielded valuable and rich information on the patient experience and perceptions of hematologic cancers, in particular for acute myeloid leukemia, and its treatments. Further understanding of these insights could aid discussions between clinicians, patients, and their caregivers regarding treatment decisions, highlight outcomes of importance to patients in clinical studies, and ultimately, inform patient-focused drug development and evaluation.

Symptom Clusters and Influencing Factors in Children With Acute Leukemia During Chemotherapy.

BACKGROUND: Acute leukemia is the most common malignancy in childhood. Identification of symptom clusters and their influencing factors in children with acute leukemia may improve the management of symptoms. OBJECTIVE: The aims of this study were to investigate symptom clusters in children with acute leukemia during chemotherapy and analyze the factors influencing their severity. METHODS: A cross-sectional survey was administered to 159 Chinese children with acute leukemia during chemotherapy. A demographic questionnaire and the Memorial Symptom Assessment Scale 10-18 were completed by the patients and their parents. Exploratory factor analysis was used to identify symptom clusters. Univariate analyses and multiple linear regression analyses were performed to analyze the factors affecting symptom clusters. RESULTS: Six clusters were identified, including gastrointestinal cluster, emotional cluster, head-related cluster, self-image disorder cluster, skin itching/irritation cluster, and somatic cluster. Factors influencing the severity of symptom clusters included age, gender, stage of chemotherapy, and medical reimbursement. CONCLUSIONS: There are multiple symptom clusters in children with acute leukemia during chemotherapy. Medical care staff should manage symptoms from the perspective of symptom cluster and carry out personalized interventions according to factors influencing these clusters. IMPLICATIONS FOR PRACTICE: The ability to predict symptom clusters in children with acute leukemia undergoing chemotherapy may assist with optimal clinical decision making in order to alleviate the symptom burden and improve children's quality of life.

[What is recommended in the treatment of acute myeloid leukemia?] / Was ist gesichert in der Therapie der akuten myeloischen Leukämie?

Acute myeloid leukemia (AML) is characterized by a malignant transformation and proliferation of myeloid progenitor cells that cause a replacement of normal hematopoiesis. Diagnostic workup for AML includes cytogenetic analysis and mutational screening covering frequently mutated genes in AML. The genetic analysis is required for risk stratification and treatment decisions. Very recently, three novel drugs have been approved for patients who can be intensively treated: a tyrosine kinase inhibitor (midostaurin) for patients with FLT3 mutations, a liposomal formulation of chemotherapy (CPX) for patients with features of secondary AML, and a CD33 antibody-drug conjugate (gemtuzumab-ozogamicin) for AML with CD33 expression. Allogeneic stem cell transplantation remains an important treatment strategy for patients with intermediate- or high-risk AML and for patients with relapsed AML. For elderly patients who cannot undergo intensive treatment, demethylating agents are the treatment of choice. The aim is to prolong life expectancy with acceptable quality of life. In recent clinical trials, novel drugs have shown promising results in this patient population. Some of these drugs have already been approved in the US. Among these drugs are the Bcl­2 inhibitor venetoclax, which is already approved in Germany for chronic lymphatic leukemia, as well as IDH1/IDH2 inhibitors (the latter for patients with IDH1/IDH2 mutated AML). Acute promyelocytic leukemia represents a special type of AML that should be treated with a combination of all-trans retinoic acid and arsenic trioxide leading to excellent outcome.

Supporting Family Caregivers of Adult and Pediatric Persons with Leukemia.

OBJECTIVES: To describe the needs and formal assessment of family caregivers and ways to intervene to alleviate distress and enhance caregiving skills in the setting of adult and pediatric leukemia. DATA SOURCES: Literature review, clinical practice observations and experiences. CONCLUSION: While rapid treatment advances in leukemia are a welcome development, the reliance on complex care delivered by family members across settings continues to grow and, concomitantly, so does the risk of mental, physical, and economic burden. IMPLICATIONS FOR NURSING PRACTICE: Oncology nurses and other clinicians should systematically incorporate screening and assessment services so that educational and referral needs are identified and intervened upon.

Psychological and Financial Distress Management in Adults With Acute Leukemia.

OBJECTIVES: To review and summarize psychological and financial distress in adults with acute leukemia and provide evidence-based methods that nurses can apply to alleviate distress experienced by patients. DATA SOURCES: Oncology and palliative care literature published over the past decade. CONCLUSION: Current evidence supports the efficacy of psychosocial interventions to improve psychological well-being and reduce distress for adults undergoing treatment for leukemia. IMPLICATIONS FOR NURSING PRACTICE: Nurses play a critical role, not only in identifying patients who are experiencing impaired psychological well-being and high levels of distress, but also in providing prompt support to these patients.

Anxiety and depression predict unfavorable survival in acute myeloid leukemia patients.

This study aimed to investigate the prevalence/severity of anxiety and depression, and also their correlations with clinical characteristics and survival profiles in acute myeloid leukemia (AML) patients.In all, 208 AML patients and 200 age and sex-matched healthy controls (HCs) were recruited in this study. Anxiety and depression were assessed by the Hospital Anxiety and Depression Scale (HADS) in AML patients before initiating therapy and in HCs after being enrolled. Treatment response was assessed, and event-free survival (EFS), and also the overall survival (OS) were calculated.The HADS-anxiety score (P < .001), anxiety prevalence (P < .001), and anxiety severity (P < .001) were increased in AML patients than those in HCs. The HADS-depression score (P < .001), depression prevalence (P < .001), and also depression severity (P < .001) were higher in AML patients compared with HCs. No correlation of anxiety or depression with clinical characteristics was found in AML patients (all P > .05). Moreover, the anxiety (P = .178) and depression (P = .512) rates were similar between complete remission (CR) patients and non-CR patients. Additionally, the EFS was worse in anxiety patients compared with nonanxiety patients (P = .013). The OS was shorter in anxiety patients compared with nonanxiety patients (P = .015) and was also worse in depression patients compared with nondepression patients (P = .007).Anxiety and depression are much more frequent and severe in AML patients compared to HCs, and both of them predict unfavorable survival profiles in AML patients.

Comparison of quality of life and health behaviors in survivors of acute leukemia and the general population.

We aimed to compare the health-related quality of life and health behaviors of acute leukemia (AL) survivors with that of the general population from two cohorts. AL survivors (n = 149) completed a set of questionnaires to evaluate quality of life, mental status, and health behaviors. AL survivors had more physical and mental difficulties (problems with usual activities, 15% vs. 5%, p < 0.001; anxiety or depression, 24% vs. 9%, p < 0.001; pain, 35% vs. 20%, p = 0.002) and more financial difficulties (p < 0.001) than the general population. Survivors who received stem cell transplantation (SCT) had significantly worse problems with role functioning, fatigue, pain, dyspnea, and insomnia, and had higher depression scores than chemotherapy group (p = 0.024). In terms of health behaviors, AL survivors had lower rates of smoking and drinking and higher influenza vaccination rates than the general population. However, only 17% of survivors had been recommended to receive screening for other cancers from health-care providers, and 67% thought their risk for other cancers was equal or lower than that of the general population. Cancer screening rates were even lower in the SCT group than in the chemotherapy group (p = 0.041). Our study indicates that clinicians should establish more appropriate survivorship care plans.

Quality of life in pediatric acute myeloid leukemia: Report from the Children's Oncology Group.

INTRODUCTION: Objectives were used to describe guardian proxy-report and child self-report quality of life (QoL) during chemotherapy for pediatric acute myeloid leukemia (AML) patients. METHODS: Patients enrolled on the phase 3 AML trial AAML1031 who were 2-18 years of age with English-speaking guardians were eligible. Instruments used were the PedsQL Generic Core Scales, Acute Cancer Module, and Multidimensional Fatigue Scale. Assessments were obtained at the beginning of Induction 1 and following completion of cycles 2-4. Potential predictors of QoL included the total number of nonhematological grade 3-4 Common Terminology Criteria for Adverse Event (CTCAE) submissions. RESULTS: There were 505 eligible guardians who consented to participate and 348 of their children provided at least one self-report assessment. The number of submitted CTCAE toxicities was significantly associated with worse physical health summary scores (ß ± standard error (SE) -3.00 ± 0.69; P < 0.001) and general fatigue (ß ± SE -2.50 ± 0.66; P < 0.001). Older age was significantly associated with more fatigue (ß ± SE -0.58 ± 0.25; P = 0.022). Gender, white race, Hispanic ethnicity, private insurance status, risk status, bortezomib assignment, and duration of neutropenia were not significantly associated with QoL. DISCUSSION: The number of CTCAE toxicities was the primary factor influencing QoL among children with AML. Reducing toxicities should improve QoL; identifying approaches to ameliorate them should be a priority.