Health-related quality of life and well-being in parents of infants and toddlers with cancer.

Background: The unique psychosocial needs of parents and caregivers of young children with cancer are poorly understood. The aims of the present study were to examine health-related quality of life (hrqol), stress, and psychological distress in parents of young children (0-4 years) diagnosed with cancer; and the associations between parent psychosocial functioning and child treatment characteristics. Methods: Parents (n = 35) with a child (n = 19 male, 54.3%) 0-48 months of age (median: 31.06 months) on active cancer therapy were recruited. Parents completed questionnaires related to demographics, parent hrqol, parenting stress, posttraumatic stress symptoms, and parent psychological distress. Results: Parents reported clinically elevated parenting stress (5.9%), posttraumatic stress symptoms (18.2%), and psychological distress (21.9%). Compared with population norms, parents reported lower hrqol in the vitality (t = 5.37, p < 0.001), mental health (t = 4.02, p < 0.001), role limitation-emotional (t = 3.52, p < 0.001), and general health perceptions (t = 2.25, p = 0.025) domains. Social functioning (ß = 0.33, p = 0.041) predicted general health perceptions; vitality (ß = 0.30, p = 0.134) and parent mental health (ß = 0.24, p = 0.285) did not [F(3,29) = 12.64, p < 0.001, R2 = 0.57]. Conclusions: A subset of parents of young children on active cancer treatment experience clinically elevated psychosocial symptoms. Having poor social connections put parents at risk of perceiving their health more poorly in general. Supports that focus on preventing the emergence of clinically significant distress should focus on parents of young children with cancer who are most at risk of poor outcomes.

Quality of life in pediatric cancer survivors: contributions of parental distress and psychosocial family risk.

BACKGROUND: Pediatric survivors of childhood cancer are at increased risk of poor quality of life and social-emotional outcomes following treatment. The relationship between parent psychological distress and child adjustment in pediatric cancer survivors has been well established. However, limited research has examined the factors that may buffer this association. The current study examined the associations between psychosocial family risk factors, parental psychological distress, and health-related quality of life (hrql) in pediatric cancer survivors. METHODS: Fifty-two pediatric cancer survivors (34 males, 18 females, mean age = 11.92) and their parents were recruited from a long-term cancer survivor clinic. Children and their parents who consented to participate completed the Pediatric Quality of Life Inventory 4.0. Parents completed a demographic information form, the Psychosocial Assessment Tool (pat 2.0) and the Brief Symptom Inventory (bsi). The Intensity of Treatment Rating (itr-3) was evaluated by the research team. RESULTS: Multiple regression analyses revealed that parental psychological distress negatively predicted parent-reported hrql, while treatment intensity, gender, and psychosocial risk negatively predicted parent and child-reported hrql. Psychosocial risk moderated the association between parent psychological distress and parent-reported child hrql (p = 0.03), whereby parents with high psychological distress but low levels of psychosocial risk reported their children to have higher hrql. CONCLUSION: Low levels of family psychosocial risk buffer the impact of parent psychological distress on child hrql in pediatric cancer survivors. The findings highlight the importance of identifying parents and families with at-risk psychological distress and psychosocial risk in order to provide targeted support interventions to mitigate the impact on hrql.

Outcomes following autologous hematopoietic stem cell transplant for patients with relapsed Wilms' tumor: a CIBMTR retrospective analysis.

Despite the marked improvement in the overall survival (OS) for patients diagnosed with Wilms' tumor (WT), the outcomes for those who experience relapse have remained disappointing. We describe the outcomes of 253 patients with relapsed WT who received high-dose chemotherapy (HDT) followed by autologous hematopoietic stem cell transplant (HCT) between 1990 and 2013, and were reported to the Center for International Blood and Marrow Transplantation Research. The 5-year estimates for event-free survival (EFS) and OS were 36% (95% confidence interval (CI); 29-43%) and 45% (95 CI; 38-51%), respectively. Relapse of primary disease was the cause of death in 81% of the population. EFS, OS, relapse and transplant-related mortality showed no significant differences when broken down by disease status at transplant, time from diagnosis to transplant, year of transplant or conditioning regimen. Our data suggest that HDT followed by autologous HCT for relapsed WT is well tolerated and outcomes are similar to those reported in the literature. As attempts to conduct a randomized trial comparing maintenance chemotherapy with consolidation versus HDT followed by stem cell transplant have failed, one should balance the potential benefits with the yet unknown long-term risks. As disease recurrence continues to be the most common cause of death, future research should focus on the development of consolidation therapies for those patients achieving complete response to therapy.

Adverse reactions during stem cell infusion in children treated with autologous and allogeneic stem cell transplantation.

Adverse reactions (ARs) during the infusion of cellular therapy products (CTPs) are common in patients undergoing hematopoietic stem cell transplantation (HSCT). We retrospectively studied pediatric patients undergoing autologous and allogeneic HSCT to determine the incidence and grade of ARs during stem cell infusion and their predictors. We analyzed data from 213 patients (120 allogeneic and 93 autologous) who received at least 1 CTP, totaling 361 infusion episodes. Serious ARs, defined as grade 2 and 3, occurred in 25 and 11% of infusions, respectively. No grade 4 or 5 ARs were noted. Independent risk factors for developing a serious AR included stem cell source (PBSC vs marrow (odds ratio (OR) 1.8, 95% confidence interval (CI): 0.4-9); cord vs marrow (OR 7.3, 95% CI: 1.3-40), overall P=0.0001) but manipulated CTPs were protective (OR 0.4, 95% CI: 0.2-0.7, P=0.004). Unlike previous adult studies, WBC and granulocyte content were not found to be risk factors in this pediatric population. These data suggest that children tolerate higher WBC content during infusion of CTPs and support the use of manipulated CTP, as indicated, to reduce the risk of adverse infusion reactions.

Single-agent high-dose melphalan followed by auto-SCT for relapsed and refractory Hodgkin lymphoma in children and adolescents.

Hodgkin lymphoma (HL) is cured in the majority of children and adolescents. However, there remains a group of patients with primary refractory or relapsed disease for whom cure is more difficult to achieve. Most of these patients receive high-dose chemotherapy followed by auto-SCT, with expected cure rates ranging from 40 to 60%. Conditioning regimens often consist of multiple non-cross-resistant agents, with well-described risks of morbidity and mortality. The use of single-agent high-dose melphalan (HDM) as conditioning, before autologous rescue, has been described in adult patients at our center, with comparable efficacy and less morbidity. We present a series of eight pediatric patients conditioned with single-agent HDM before autologous stem cell rescue for relapsed and primary refractory HL. All patients engrafted with a median of 12 days to neutrophil engraftment. Two patients subsequently relapsed. Seven patients are currently alive, and seven of eight patients have no evidence of disease (one in CR3). Toxicities included grade 4 hematologic in 8/8, grade 3 mucositis in 3/8, grade 3 infectious in 2/8 and grade 4 infectious in 1/8. Our analysis suggests that this regimen is feasible in pediatric patients with acceptable engraftment and toxicity.

A questionnaire based review of long-term follow-up programs for survivors of childhood cancer in Canada.

BACKGROUND: Major advances have been made in the treatment of childhood cancer; however, survivors of childhood cancer are at increased risk for morbidity and mortality. There is little literature regarding available long-term follow-up programs for survivors of childhood cancer. PROCEDURE: In March 2007, 16 surveys were sent to pediatric hematology/oncology programs across Canada to determine what programs were available for survivors of childhood cancer, and the nature of such programs. RESULTS: Of 15 participating centers, 13 (87%) have multi-disciplinary programs for the long-term follow-up of pediatric cancer survivors. Research databases were documented in 9/15 (60%) of centers to document late effects. Dedicated programs for adult survivors of childhood cancer were established in 8/15 (53%) of centers. Access to subspecialty care for survivors was rated as quite good. Concerns were raised by many participants about patients being lost to follow-up. Respondents indicated that primary care physicians appear to be under-represented within dedicated long-term follow-up programs. CONCLUSION: Long-term follow-up programs for survivors of childhood cancer are available in 87% of Canadian pediatric oncology centers. While programs reported good access to care for childhood survivors, many adult survivors of childhood cancer have more limited timely access to services and patients are often lost to follow-up. New models of care incorporating primary care physicians are necessary due to growing numbers of survivors.

Treatment of intracranial and extracranial haemorrhages in a neonate with severe haemophilia B with recombinant factor IX infusion.

Intracranial (ICH) and extracranial (ECH) haemorrhages are potentially life-threatening events that may occur comorbidly in neonates with haemophilia. There is little data on the use of recombinant factor IX (rFIX; BeneFIX in the neonate. Children <15 years of age are known to require higher doses of recombinant Factor IX (FIX) than older persons, which raises specific concerns in the neonate due to the increased risk of thrombosis in this age group (Thromb Haemost 2002; 87: 431). This report describes a case in which a high rate of continuous infusion of recombinant FIX was used to treat a newborn with significant intracranial and subgaleal haemorrhages. A high rate of infusion maintained at 30-35 U kg(-1) h(-1) was necessary to maintain adequate FIX levels. Despite the high rate of continuous infusion, no adverse events were noted. Our patient had a rare genetic mutation causing severe haemophilia B. A neonate with severe haemophilia B was treated successfully with recombinant FIX through continuous infusion. A high rate of infusion was required and no complications were noted.