Amy Garee

Establishing psychosocial palliative care standards for children and adolescents with cancer and their families: An integrative review

Background: Despite standardization in disease assessments and curative interventions for childhood cancer, palliative assessments and psychosocial interventions remain diverse and disparate. Aim: Identify current approaches to palliative care in the pediatric oncology setting to inform development of comprehensive psychosocial palliative care standards for pediatric and adolescent patients with cancer and their families. Analyze barriers to implementation and enabling factors. Design: Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines framed the search strategy and reporting. Data analysis followed integrative review methodology. Data sources: Four databases were searched in May 2014 with date restrictions from 2000 to 2014: PubMed, Cochrane, PsycINFO, and Scopus. A total of 182 studies were included for synthesis. Types of studies included randomized and non-randomized trials with or without comparison groups, qualitative research, prior reviews, expert opinion, and consensus report. Results: Integration of patient, parent, and clinician perspectives on end-of-life needs as gathered from primary manuscripts (using NVivo coding for first-order constructs) revealed mutual themes across stakeholders: holding to hope, communicating honestly, striving for relief from symptom burden, and caring for one another. Integration of themes from primary author palliative care outcome reports (second-order constructs) revealed the following shared priorities in cancer settings: care access; cost analysis; social support to include primary caregiver support, sibling care, bereavement outreach; symptom assessment and interventions to include both physical and psychological symptoms; communication approaches to include decision-making; and overall care quality. Conclusion: The study team coordinated landmark psychosocial palliative care papers into an informed conceptual model (third-order construct) for approaching pediatric palliative care and psychosocial support in oncology settings.

Palliative Care as a Standard of Care in Pediatric Oncology.

The study team conducted a systematic review of pediatric and adolescent palliative cancer care literature from 1995 to 2015 using four databases to inform development of a palliative care psychosocial standard. A total of 209 papers were reviewed with inclusion of 73 papers for final synthesis. Revealed topics of urgent consideration include the following: symptom assessment and intervention, direct patient report, effective communication, and shared decision‐making. Standardization of palliative care assessments and interventions in pediatric oncology has the potential to foster improved quality of care across the cancer trajectory for children and adolescents with cancer and their family members. Pediatr Blood Cancer

Sorafenib treatment following hematopoietic stem cell transplant in pediatric FLT3/ITD acute myeloid leukemia

FLT3/ITD is associated with poor outcomes in adult and pediatric acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation (HSCT) can improve cure rates, however relapse is still common. Recent studies demonstrate the activity of FLT3 inhibitors, including sorafenib, in targeting the underlying mutation.

Metabolic syndrome and endocrine dysfunctions after HSCT in children

MS and endocrine dysfunction(s) are common well‐recognized complications after HSCT. We retrospectively analyzed our data on 160 patients with a median age at transplant of five yr (0.3–23), who had been followed for a median of seven yr (range 3–18) at Nationwide Childrens Hospital after transplant. Dyslipidemia and MS were seen in 13% and 7.5% patients, respectively, and 58% of these patients were <20 yr of age. Twelve patients met the criteria for diagnosis of MS, but four of these did not meet the International Diabetic Federation or WHO criteria. Variation in the diagnostic criteria for MS leading to underdiagnosis is discussed. Female gonadal failure (27%) and hypothyroidism (21%) were the most common endocrine dysfunctions, followed by short stature and GH deficiency (17%) each. TBI and younger age at HSCT were associated with the highest burden of long‐term effects, and female sex was more significantly associated with MS‐related dysfunction (p < 0.05). Uniform diagnostic criteria for MS and close follow‐up after transplant are important for the early diagnosis and management of these late effects, thereby improving the overall quality of life of these patients.

Disseminated Rhizomucor pusillus causing early multiorgan failure during hematopoietic stem cell transplantation for severe aplastic anemia.

Matched sibling donor hematopoietic stem cell transplantation is the standard of care for severe aplastic anemia, with an overall survival of 80% to 90%. Only 60% to 70% of patients respond to treatment with immunosuppressive therapy. The main life threatening complications are infections, graft failure, and graft versus host disease. A 10-year-old patient with severe aplastic anemia underwent matched sibling donor hematopoietic stem cell transplantation, but developed sudden onset of fatal multiorgan failure on day +12. The cause of death was found only after autopsy.

Impact on Care of the Bone Marrow Transplant Patient With the Utilization of Twenty-Four Hour Coverage by Nurse Practitioners: 21

PBMTC Fall Conference September 19–21, 2005 01. Umbilical Cord Blood Transplantation From Related and Unrelated Donors in Children Patients A. Ghavamzadeh, K. Alimoghaddam, A.A. Hedayatiasl, A. Mousavi, M. Iravani, B. Bahar, M. Jahani, M. Faranoush, A.R. Shamshiri. HematologyOncology and Bone Marrow Transplantation Research Center Tehran University of Medical Science. Introduction: Cordblood banks have increased the use of cord blood transplantation (CBT) in patients with hematologic, non-hematologic and malignant disorders. Cord blood transplantation may lower the risk of graftversus –host disease (GVHD). Patients & Methods: Ten patients (Pts) with Thalassemia (7), AML (1), Hurler’s (1), SCID (1) received cord blood transplantation from their HLAidentical siblings and unrelated donors. Age range was 8mo-14 yrs (M/F = 8/2, median age 5.5 years). Eight Pts received cord blood stem cell from siblings and 2 Pts received from unrelated donors. There were no mismatches in (6) and there were mismatches of one HLA antigen in (2) and two HLA antigens in (2) Pts (unrelated donors). Conditioning regimens consisted Cyclophosphamide, Busulfan (+ATG for unrelated donors). GVHD prophylaxis regimen was Cyclosporine A6 Methotrexate(MTX). Results: Five Pts engrafted (one patient received MTX in GVHD prophylaxis and 4 Pts no received MTX). Five Pts had graft failure (all of them received MTX in GVHD prophylaxis). The median volume collected was 69 mL (range 25–110), the median number of WBC was 2.913 10 (range 0.93 10– 10.623 10), MNC was 2.083 10 (0.453 10–5.843 10) and CD34 was 1.763 10 (range 0.093 10–9.53 10). The median number of days required for neutrophil count .0.53 10/L was 21 (range 14–48) and platelet count of .203 10/L was noticed on day 60 (range 26–70). Two patients which transplanted with unrelated CB had aGVHD grade III and one patient which transplanted with related CB had aGVHD grade I and no cGVHD. Conclusion: Cord blood is a feasible alternative source of HSCT for Pts with hematologic disorders. Recipients of cord blood transplant from HLAidentical siblings have a lower incidence of acute and chronic GVHD than recipients of bone marrow transplants from HLA-identical siblings. Use of MTX for GVHD prophylaxis may associate with a greater risk of graft failure. 02. Allogeneic Blood and Marrow Transplantation in Fanconi Anemia With Fludarabine Based Conditioning Regimen A. Ghavamzadeh, M. Iravani, M. Jahani, K. Alimoghadam, A.A. Hedayatiasl, G.R. Bahoush, A. Mousavi, B. Bahar. Hematology-Oncology and Bone Marrow Transplantation Research Center Tehran University of Medical Sciences. Background: Fanconi anemia (FA) is an autosomal recessive disorder, usually characterized by several congenital malformations, progressive bone marrow failure and an increased incidence of malignancies. Cells from FA patients are hypersensitive to DNA crosslinking agents, which are used as a diagnostic tool. Methods: Because 2002, six patients (Pts) with Fanconi Anemia received blood and marrow transplantation from their HLA-identical sibling. Age range was 6–18 yrs (M/F = 4/2, median age 10.5 years). 5 patients received peripheral blood stem cell and 1 patient received bone marrow transplantation. They received Fludarabine 30 mg/m/IV (from day -9 to -5), ATG 10 mg/kg/IV (from day -4 to -1) and Cyclophosphamide 10 mg/kg/IV (from day -4 to -3) in preparative regimen. GVHD prophylaxis regimen was Cyclosporine A+ Methotrexate. Results: Median time of absolute neutrophil count .0.5 3 10/L was +10 (Range 5–13 days) and platelet count of 203 10/L was noticed on day + 12 (Range 5–24 days). Engraftment was demonstrated by STR_PCR analysis. Two patients developed aGVHD (grade I = 1, grade II = 1). At present 5 out of 6 are alive and disease free. One patient died due to rejection and pneumonia. No patient developed cGVHD. Conclusions: Allogeneic blood and marrow transplantation with this regimen is a suitable treatment of Fanconi anemia. More assessment is needed for evaluating of this regimen. 03. Allogeneic Bone Marrow Transplantation Following Conditoning With Busulphan and Melphalan Jairam Sastry, MBBS, MD, MRCP, MRCPCH, Peter J. Shaw, MA, MBBS, MRCP, FRACP. The Children’s Hospital at Westmead Sydney, NSW, Australia. Introduction: The outcome of treatment of AML in children is improving steadily. Allogeneic BMT still provides the best control of AML, but limited by excess mortality, related mainly to the complications of the conditioning regimen, infection and GVHD. We report the outcome and toxicity of a BU/MEL combination conditioning in children undergoing allogeneic BMT for AML. Methods: 7 children who under went allogeneic BMT for AML following conditioning with BU/MEL. Retrospective analysis of case notes and unit databases was done to collect data. Results: Six of the seven patients showed WBC engraftment. The median time of recovery to WBC

Toward a Better Understanding of Pediatric BMT Adherence: An Exploration of Provider Perceptions

1.0 3 10/L and absolute neutrophil count (ANC) to

An Investigation of the Pediatric Bone Marrow Transplant Nurses Perception of Palliative Care

0.5 3 10/L for 3 consecutive days was 14 days (range = 8–20 days) and 15 days (range = 9–25 days) respectively. Five of the 7 patients showed platelet engraftment. The median time of recovery to platelet count of

Impact of Specialty Nursing Model On Pediatric Bone Marrow Transplant Patient Outcomes

20 3 10/L and of

Vitamin D Status and its Correlation with Bone Mineral Density in Long Term Survivors After Childhood Hematopoietic Stem Cell Transplantation

50 3 10/L for 3 consecutive days was 20 days (range = 13–37 days) and 46 days (range = 17–79 days) respectively. Acute GVHD was seen in 4 of the 7 patients (Gr I –1, Gr II –3). Only one patient developed chronic GVHD. Three patients died; two from relapse and one from non-engraftment and multiorgan failure. The complications included profound myelosuppression, Mucositis, VOD of liver. Conclusions: The combination of Bu/Mel was found to be effective in terms of engraftment. The complications were significant but tolerable. This combination requires further large multicenter trial against BU/CY. A case control study comparing BU/MEL with BU/CY was proposed to the IBMTR. In the absence of such a study, we will continue to explore the use of combination of BU/MEL in patients who are not standard risk AML. 05. Late Effects of Stem Cell Transplantation Compared to Chemotherapy/Radiation Reported by Survivors in a Web-based Survey Yvonne J. Barnes, RN MSN CPNP, Marnie Hauff , Shalini Shenoy, MD. St. Louis Childrens Hospital, St. Louis, Missouri and Washington University School of Medicine, St. Louis, Missouri. Background: Intensity and duration of chemotherapy and/or radiation delivered for the treatment of cancer dictates toxicities. Age at diagnosis, tumor, location, surgical intervention and stem cell transplantation (SCT) are additional factors that influence complications. Aim: Analyze toxicities perceived by survivors after SCT versus without using a web-based survey. 460 q 2005 Lippincott Williams & Wilkins Methods: A project by the Late Effects Program at our institution included a summary of side effects post therapy on our website. The goal was to disseminate basic information, raise medical awareness, list resources and encourage follow up. An anonymous survey invited visitors to report treatment and describe late effects. Survey questions included diagnosis, age, treatment, duration, medical/psychosocial complications and adequacy of care perceived by the respondent. Results: Twenty-two SCT survivors responded, including parents (19), patient (1) or friends (2). Median age at diagnosis was 2–5 years. Sixteen had completed therapy ,5years, 3 were 5–10 years post and 3 unknown. There were 7 male and 9 females (6 unknown). Diagnoses included leukemia (7), solid tumors (12), lymphoma (2) and other (2). Conditioning for SCT included radiation in 13 (59%). Overall, 63.6% (14/22) reported adequate medical support. Conclusions: SCT recipients were 1.2 times more likely to experience late effects. Growth failure, delayed puberty, infertility, cosmetic, dental and endocrine problems were increased following SCT. Radiation and chemotherapy survivors described more learning disabilities. Both groups had rare patients (2) that perceived no effects. The high incidence of late effects ranging from medical issues to psychosocial underscores the need for Late Effects multidisciplinary clinics. 06. Allogeniv HLA Identical Bone Marrow Transplantation in Patient With Juvenile Idiopathic (Chronic) Arthritis (JIA) Kálmán Nagy, Gabriella Marton, Gabriella Márton, Sándor Sipka. Borsod University Hospital Pediatric BMT Unit, Miskolc, Hungary, DEOEC III. Department of Internal., Debrecen, Hungary. Autologous stem cell transplantation (ASCT) is considered a promising treatment modality of autoimmun disease. 5 years ago we performed a CD 34 selected autologous stem cell transplantation in a patient with JIA. The patient had new flare six months after ASCT. The next patient received allogenic graft. 9-year-old girl with systemic form of JIA had a six year history of swelling and stiffnes of small joints of hands, wrists, temporomandibular joint and feet to deformity and disability. Her arthritis was poorly controlled and she had very high inflammatory activity. She received corticosteroid, immunosuppresssive and biologic treatment (oral and pulse cyclophosphamid, methotrexate, etanercept). The patient had an HLA identical brother and we decided to perform allogenic bone marrow transplantation. Conditioning regimen Busulfan 16 mg/kg, Cyclophosphamid 120 mg/kg, ATG/Fresenius/40 mg/kg. Allogenic BMT was performed 25. sept. 2003. The patient received bone marrow mononuclear cells 2,4 3 10 kg bw recipient. She engrafted at the postransplant day +13. VNTR examination demonstrated only donor original cells in peripheral blood at +51 posttransplant day. Proinflammatoric and inflammatoric cytokine levels: Pretransplant: TNF alfa: 146,4, IL-1: 82,13, IL-2: 21,04, IL-6: 47,79 pg/ml, posttransplant 3 month: TNF alfa: 0, IL-l: 11,09, IL-2: 21,04, Il-6: 20,5 pg/ml, posttransplant: 6 month: TNF alfa: 0,23, IL-2: 1,8, IL-6: 1.91 pg/ml, posttransplant 9 month: TNF alfa: 0, IL-1: 0,74, IL-2: 0 pg/ml. 21 months after the transplantation the patient is well w