Treatment Pattern and Outcomes in Newly Diagnosed Multiple Myeloma Patients Who Did Not Receive Autologous Stem Cell Transplantation: A Real-World Observational Study : Treatment pattern and outcomes in patients with multiple myeloma.

INTRODUCTION: The objective of this study was to describe the treatment patterns among patients with newly diagnosed multiple myeloma (MM) who had not received autologous stem cell transplantation (ASCT). It further compares the safety and clinical outcomes across different frontline regimens as well as explores whether treatment duration predicts outcomes. METHODS: Patients with MM (> 45 years) who had not received ASCT were retrospectively identified from the US SEER-Medicare (Jan 2007-Dec 2016) and Optum (Jan 2007-Sep 2018) databases. Cox proportional hazard models were used to compare overall survival (OS) among bortezomib + lenalidomide + dexamethasone regimen (VRd), lenalidomide + dexamethasone regimen (Rd), cyclophosphamide + bortezomib + dexamethasone regimen (CyBorD), bortezomib + dexamethasone regimen (Vd), and other bortezomib-containing therapies based on propensity score matching. To address immortal time bias, time-fixed and time-dependent Cox models were employed to estimate the association of longer frontline treatment exposure with outcomes. RESULTS: Mean (standard deviation; SD) age was 71 (9.8) years; and 49.51% were women. Bortezomib and lenalidomide-based combinations were the most common treatment modalities. After matching, the HR (95% CI) of OS by frontline therapies comparing VRd with Vd was 0.76 (0.66, 0.86), CyBorD was 0.87 (0.75, 1.05), for other bortezomib-based therapies was 0.56 (0.49, 0.64), Rd was 0.83 (0.73, 0.95), and for other therapies was 0.70 (0.61, 0.80). Longer frontline treatment duration was associated with better OS for overall frontline [HR (95% CI) 0.86 (0.82, 0.90)]; Vd [0.81 (0.74, 0.89)]; CyBorD [0.79 (0.64, 0.98)] and Rd [0.86 (0.78, 0.95)]. CONCLUSION: Results demonstrated that the frontline therapies prescribed to most patients who did not receive ASCT for MM in the United States were consistent with the NCCN guideline recommendations. Longer frontline treatment duration was associated with improved OS.

Optimal care for the child with cancer: A summary statement from the SIOP Working Committee on Psychosocial Issues in Pediatric Oncology.

Since its foundation in 1991, the SIOP Working Committee on Psychosocial Issues in Paediatric Oncology1 has developed and published 12 sets of Guidelines for health-care professionals treating children with cancer and their families. Those elements considered essential in the process of cure and care of children with cancer are summarized in this document as a formal statement, developed at the 2007 SIOP annual meeting in Mumbai. Elaboration of the concepts with detailed strategies for practice can be found in the referenced guidelines [1-12] and in a companion publication [13]. This article is a summary of what practitioners considered critical elements in the optimal care of the child with cancer, with the goal of stimulating a broader application of these elements throughout the SIOP membership.

Safety of summer camp for children with chronic and/or life threatening illness.

The purpose of this work was to retrospectively evaluate the medical needs of children with chronic and/or life-threatening illness attending summer camp at Barretstown Gang Camp (Ireland). Data on medical facility visits collected over 28 sessions (2120 children) between 1998 and 2001 were reviewed. Children originated from 20 different European countries. The most common diagnoses were leukaemia, lymphoma, sarcoma and brain tumour. Forty-eight percent of the children required medical care during their stay and 3386 total visits were recorded. The most common cause of medical facility visit was pain, followed by injury (trauma, bruises, burn) and flu/cold symptoms. Only 8 brief hospital transfers were necessary for the 2120 children. This large-scale study confirms the safety of a well-organized medically supervised summer therapeutic recreational program for children with chronic conditions, including children undergoing chemotherapy treatment and factor replacement.

Patterns of failure in supratentorial primitive neuroectodermal tumors treated in Children's Cancer Group Study 921, a phase III combined modality study.

PURPOSE: To analyze the patterns of failure in patients with supratentorial primitive neuroectodermal tumors (ST-PNETs) treated with combined modality therapy in a large, randomized, multi-institutional study. METHODS AND MATERIALS: A total of 44 prospectively staged patients with ST-PNET confirmed by central pathology review were treated in the Children's Cancer Group Study 921, which compared two chemoradiotherapy regimens. The patterns of initial sites of failure were analyzed. These were compared with the failure patterns of 188 children with posterior fossa (PF) PNETs treated in the same protocol. RESULTS: The major determinant for progression-free survival was the initial metastatic stage. The 3-year progression-free survival for M0 patients was 53% +/- 8.5% compared with 14% +/- 9.4% for M+ patients. The cumulative 5-year relapse incidence was 71.4% +/- 21% for M+ patients compared with 47.5% +/- 8.6% for M0 patients. The overall failure rate for both M0 and M+ ST-PNETs was greater than that for PF-PNETs (47.5% +/- 8.6% vs. 29.3% +/- 4.7% for M0 and 71.4% +/- 21% vs. 48.4% +/- 5.5% for M+). Failure at the primary site, either as the sole site or as a component of initial failure, was also seen more frequently in ST-PNETs than in PF-PNETs. For M0 patients, the 5-year local failure rate as a component of initial failure was 42.0% +/- 8.5% for ST-PNETs compared with 17.7% +/- 3.9% for PF-PNETs. For patients with primary tumors either in the ST or PF, the 5-year spinal axis failure rate as a component of initial failure was not significantly different statistically when compared by M stage. For M+ patients, the 5-year spinal axis failure rate as a component of initial failure was 42.9% +/- 22.8% for ST-PNETs and 34.6% +/- 5.2% for PF-PNETs. CONCLUSION: Despite aggressive combined modality therapy, ST-PNETs had high rates of failure, with M+ patients faring especially poorly. Both local and spinal failure rates remained high, indicating the need to maximize both local and regional/systemic therapies. Overall, these patients fared worse than those with high-risk PF-PNETs in terms of progression-free survival and failure rates.

Initial evaluation of an electronic symptom diary for adolescents with cancer.

BACKGROUND: The delivery of optimal care depends on accurate communication between patients and clinicians regarding untoward symptoms. Documentation of patients' symptoms necessitates reliance on memory, which is often imprecise. We developed an electronic diary (eDiary) for adolescents and young adults (AYAs) with cancer to record symptoms. OBJECTIVE: The purpose of this paper is to describe the utility of an eDiary designed for AYAs with cancer, including dependability of the mobile application, the reasons for any missing recorded data, patients' adherence rates to daily symptom queries, and patients' perceptions of the usefulness and acceptability of symptom data collection via mobile phones. METHODS: Our team developed an electronic symptom diary based on interviews conducted with AYAs with cancer and their clinicians. This diary included daily severity ratings of pain, nausea, vomiting, fatigue, and sleep. The occurrence of other selected physical sequelae was assessed daily. Additionally, patients selected descriptors of their mood. A 3-week trial of the eDiary was conducted with 10 AYA cancer patients. Mobile phones with service plans were loaned to patients who were instructed to report their symptoms daily. Patients completed a brief questionnaire and were interviewed to elicit their perceptions of the eDiary and any technical difficulties encountered. RESULTS: Overall adherence to daily symptom reports exceeded 90%. Young people experienced few technical difficulties and reported benefit from daily symptom reports. Symptom occurrence rates were high and considerable inter- and intra-patient variability was noted in symptom and mood reports. CONCLUSIONS: We demonstrated the utility of an eDiary that may contribute insight into patients' symptom patterns to promote effective symptom management.

Medical utilization by immunodeficient, hemophiliac, and HIV positive children during summer camp: evidence for a safe camper experience.

BACKGROUND: Continuation of normal activities is vital to psychosocial development of children with serious illnesses. The purpose of this study was to determine whether or not it was safe for HIV-positive children and children with other immunodeficiencies to attend camp. PROCEDURE: The study population consisted of HIV (+) children, HIV negative siblings, and other immunodeficient campers attending Barretstown Gang (BG) Camp between 1998 and 2002. Their visit frequency to the on-site medical facility was compared within the study population and between 2,323 contemporaneous campers with cancer. RESULTS: Over half of the HIV (+) children were on active therapy. Greater than 97% of staff (49/51) made at least one visit compared with 64% (149/233) of campers (P < 0.04). HIV (-) siblings had almost the same need for medical attention (total visits) as children with immunodeficiencies (P = 0.34). Most visits [88%] among all diagnostic groups except hemophilia were non-disease related (328 vs. 47). Apart from URIs, there were few other infections and no fevers in the HIV(+) or immunodeficiency group, nor were there significant bleeds in the hemophiliacs. Most visits were for routine camp-type ailments. CONCLUSIONS: Our findings suggest that it is safe for HIV (+) and immunodeficient children to attend a properly staffed camp.

Patterns of treatment failure in infants with primitive neuroectodermal tumors who were treated on CCG-921: a phase III combined modality study.

PURPOSE: To analyze patterns of treatment failure in infants with primitive neuroectodermal tumors (PNETs) who were treated primarily with chemotherapy in a large multi-institutional study. MATERIALS AND METHODS: Sixty-five prospectively staged patients with PNET confirmed by central pathology review, who were 18 months or younger were treated on Children's Cancer Group Study 921 (CCG-921) primarily with chemotherapy. Forty-six patients had posterior fossa (PF) primary tumors and 19 patients had supratentorial (ST) primaries. Patterns of sites of initial treatment failure were analyzed and compared to failure patterns of 180 older children who had PF-PNETs, and 44 older children with ST-PNETs who were treated on the same protocol. RESULTS: The entire cohort of younger patients fared much worse than those older than 18 months. Cumulative 5-year relapse incidence (+/-SE) for younger patients with PF-PNETs was 64.5 +/- 8.9% for patients without metastases (M0) compared to 71.4 +/- 13.4% for patients with metastases (M+). The cumulative 5-year relapse incidences for younger patients with ST-PNETs were 64.3 +/- 13.7% for M0 patients compared to 100 +/- 33.3% for M+ patients. Relapses in these patients occurred within 2 years. The overall treatment failure rate was significantly higher for younger compared to older patients with PF-PNET and ST-PNET. There was no statistically significant difference in relapse patterns between patients with PF primary tumors and ST primaries when stratified by stage. There was no statistically significant difference in relapse patterns between M0 and M+ patients. All patients had a high risk of recurrence at primary tumor site. Younger patients who had PF primary tumors without metastasis at presentation were significantly more likely to relapse in PF than older patients. Younger patients were at significant risk of relapse in the spine, but none had it as the sole site of first relapse. CONCLUSIONS: Despite aggressive chemotherapy, younger children with PNETs have high rates of treatment failure and fare worse than high-risk, older patients with PF-PNETs, indicating the need to maximize local, regional, and systemic therapies.

Desferoxamine (DFO)--mediated iron chelation: rationale for a novel approach to therapy for brain cancer.

Iron homeostasis is crucial to normal cell metabolism, and its deficiency or excess is associated with numerous disease states. The association of increased iron load with cancer may be due to several factors including free radical production, reduction of the body's protective mechanism to combat oxidative stress, inhibition of immune systems, inhibition of essential nutrient functions, facilitation of cancer growth, suppression of antitumor actions of macrophages, and lowering of the ratio of T4-T8 positive lymphocytes. Antiproliferative effects of desferoxamine (DFO) both in vitro and in vivo are mediated by an intracellular pool of iron that is necessary for DNA synthesis rather than prevention of iron uptake from transferrin. Several clinical studies have shown it to have antitumor activity in the treatment of neuroblastoma, leukemia, bladder carcinoma, and hepatocellular carcinoma. Human neural tumor cells are susceptible to the effects of DFO. Continued study of DFO is necessary to further elucidate its antineoplastic profile and its use as an adjunct to current chemotherapy regimens. Given the lack of satisfactory treatment of central nervous system neoplasms, DFO could serve as an important tool in the management of such cancers.