Epidemiology of acute myeloid leukemia: Recent progress and enduring challenges.

Importance Patients with acute myeloid leukemia (AML) receiving intensive chemotherapy experience substantial decline in their quality of life (QOL) and mood during their hospitalization for induction chemotherapy and often receive aggressive care at the end of life (EOL). However, the role of specialty palliative care for improving the QOL and care for this population is currently unknown. Objective To assess the effect of integrated palliative and oncology care (IPC) on patient-reported and EOL outcomes in patients with AML. Design, Setting, and Participants We conducted a multisite randomized clinical trial of IPC (n = 86) vs usual care (UC) (n = 74) for patients with AML undergoing intensive chemotherapy. Data were collected from January 2017 through July 2019 at 4 tertiary care academic hospitals in the United States. Interventions Patients assigned to IPC were seen by palliative care clinicians at least twice per week during their initial and subsequent hospitalizations. Main Outcomes and Measures Patients completed the 44-item Functional Assessment of Cancer Therapy–Leukemia scale (score range, 0-176) to assess QOL; the 14-item Hospital Anxiety and Depression Scale (HADS), with subscales assessing symptoms of anxiety and depression (score range, 0-21); and the PTSD Checklist–Civilian version to assess posttraumatic stress disorder (PTSD) symptoms (score range, 17-85) at baseline and weeks 2, 4, 12, and 24. The primary end point was QOL at week 2. We used analysis of covariance adjusting and mixed linear effect models to evaluate patient-reported outcomes. We used Fisher exact test to compare patient-reported discussion of EOL care preferences and receipt of chemotherapy in the last 30 days of life. Results Of 235 eligible patients, 160 (68.1%) were enrolled; of the 160 participants, the median (range) age was 64.4 (19.7-80.1) years, and 64 (40.0%) were women. Compared with those receiving UC, IPC participants reported better QOL (adjusted mean score, 107.59 vs 116.45;P = .04), and lower depression (adjusted mean score, 7.20 vs 5.68;P = .02), anxiety (adjusted mean score, 5.94 vs 4.53;P = .02), and PTSD symptoms (adjusted mean score, 31.69 vs 27.79;P = .01) at week 2. Intervention effects were sustained to week 24 for QOL (β, 2.35; 95% CI, 0.02-4.68;P = .048), depression (β, −0.42; 95% CI, −0.82 to −0.02;P = .04), anxiety (β, −0.38; 95% CI, −0.75 to −0.01;P = .04), and PTSD symptoms (β, −1.43; 95% CI, −2.34 to −0.54;P = .002). Among patients who died, those receiving IPC were more likely than those receiving UC to report discussing EOL care preferences (21 of 28 [75.0%] vs 12 of 30 [40.0%];P = .01) and less likely to receive chemotherapy near EOL (15 of 43 [34.9%] vs 27 of 41 [65.9%];P = .01). Conclusions and Relevance In this randomized clinical trial of patients with AML, IPC led to substantial improvements in QOL, psychological distress, and EOL care. Palliative care should be considered a new standard of care for patients with AML. Trial Registration ClinicalTrials.gov Identifier:NCT02975869

/pdf/effectiveness-of-integrated-palliative-and-oncology-care-for-3lq6u00od3.pdf

Effectiveness of Integrated Palliative and Oncology Care for Patients With Acute Myeloid Leukemia: A Randomized Clinical Trial.

The aim of this study was to review and compare types of reimbursement recommendations for orphan drugs issued by eight European health technology assessment (HTA) agencies and the reimbursement status of these drugs in the corresponding countries. Separate calculations were also performed for three sub-groups: ultra-orphan drugs, oncology orphan drugs and other (non-ultra, non-oncology) orphan drugs. We reviewed drugs authorized by the European Medicine Agency (EMA) between 1 November 2002 and 30 September 2015. Among these, we identified 101 orphan drugs. Seventy-nine of them were assessed by eight European HTA agencies. The average rates of positive, conditional and negative reimbursement recommendations issued by these agencies were 55.7 %, 15.3 % and 29.0 %, respectively. On average, 21.2 % of EMA-authorized orphan drugs were reimbursed in the eight European countries studied: 49.0 % of those with positive, 53.6 % of those with conditional, and 16.0 % of those with negative reimbursement recommendations. In addition, 5.4 % of orphan drugs that had not been assessed by any of the eight HTA agencies were also reimbursed. The shares of oncology, ultra, and other orphan drugs that were assessed by HTA agencies were similar, with the lowest share observed in ultra-orphan drugs (72 %) and the highest in other orphan drugs (80 %). In terms of reimbursement, 20 % of oncology orphan drugs, 25 % of ultra-orphan drugs and 21 % of other orphan drugs were reimbursed. Reimbursement of orphan drugs does not always correspond to the type of HTA recommendation. While the highest rate of reimbursement is observed (unsurprisingly) among drugs with positive or conditional recommendation, a high rate of reimbursement (11 %) is also observed among ultra-orphan drugs that had never been assessed by any HTA agency.

/pdf/the-correlation-between-hta-recommendations-and-4nzhwkdoof.pdf

The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe

REVIEW QUESTION/OBJECTIVE The objective of this review is to present the best available evidence related to the treatment of osteosarcoma with mifamurtide in addition to standard chemotherapy. The specific review question to be addressed is: What effect does mifamurtide have on event-free survival, overall survival, and quality of life as an adjunct to chemotherapy for high-grade non-metastatic and metastatic osteosarcoma patients? INCLUSION CRITERIA Types of participants This review will consider any quantitative studies that include patients with high-grade, resectable, non-metastatic and metastatic osteosarcoma based on histological diagnosis. Patients with other types of bone cancer such as Ewing’s sarcoma and Chondrosarcoma will be excluded from this review. Types of interventions This review will consider studies that evaluate intravenous infusion of either of the pharmaceutical formulations of mifamurtide in addition to standard multi-drug chemotherapy (such as combinations of cisplatin, doxorubicin, high-dose methotrexate, and ifosfamide) after macroscopically complete surgical resection. The standard protocol for delivery of mifamurtide is 2 mg/m2 infused during a one-hour period twice a week for 12 weeks, then once a week for 12-24 weeks. Types of comparator(s) The comparison group for this review will be patients with high-grade, resectable, non-metastatic and metastatic osteosarcoma, who have received standard chemotherapy alone. Types of outcomes Primary outcomes to be considered for this review include: Event-free survival measured in years post-intervention from the time of study commencement until any recurrence or tumor progression of osteosarcoma. Overall survival of patients measured in percentage of patients surviving post-intervention with the time TRUNCATED AT 250 WORDS

/pdf/effectiveness-of-mifamurtide-in-addition-to-standard-39j1ormz3m.pdf

Effectiveness of mifamurtide in addition to standard chemotherapy for high-grade osteosarcoma: a systematic review.

Acute myeloid leukemia (AML) is a costly disease, and its impact is greater in developing countries (DC). We will review the current concept of what are DC, compare the differences in the epidemiology and economic burden of this disease between developed and DC, and finally, analyze the barriers and possible solutions that DC should implement to achieve better results. DC is a frequently misunderstood name. The way we use to measure human development is changing, and multidimension metrics better define what are DC. With this in mind, we show the differences in the AML epidemiology and the impact of economic burden in DC. We analyze the barriers to access therapy from a clinician point of view, to show that most DC shared similar challenges but with a diverse healthcare structure. Finally, we provide several possible solutions for a more integrated and timely treatment that allows better results not only in terms of survival but with a better quality of life. The economic burden of AML treatment in DC is high, and the results are poor. It is crucial to face this challenge and propose new treatment approaches to achieve better results.

/pdf/access-to-therapy-for-acute-myeloid-leukemia-in-the-2emwigmocx.pdf

Access to Therapy for Acute Myeloid Leukemia in the Developing World: Barriers and Solutions.

Systematic reviews of economic burden and health-related quality of life in patients with acute myeloid leukemia

Background
Clinical trials for treatments indicated for orphan diseases may be limited due to the low prevalence of such diseases; this can result in implications for both regulatory and health economic perspectives. This study assessed the pivotal clinical evidence packages submitted to support applications for European Medicines Agency (EMA) marketing authorizations for treatments for orphan conditions, in relation to the size of the eligible patient population.

/pdf/review-and-comparison-of-clinical-evidence-submitted-to-1n5v9x1t5q.pdf

Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatments

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Systematic reviews of economic burden and health-related quality of life in patients with acute myeloid leukemia

Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatments