Elisha Waldman

Palliative care for children with cancer.

Over the past two decades, paediatric palliative care has emerged as both a primary approach and as its own medical subspecialty, the overall aim of which is to ease suffering for children with life-threatening illness and their families through a concurrent model of care. However, most discussions have been focused on the transition to palliative care when no realistic hope for cure exists. We believe that, because the course of cancer is so unpredictable, this idea is misleading. Indeed, palliative care is increasingly being recognized as being about not just how to cope with the process of dying, but also about how to engage in living when faced with a life-threatening illness. This article will examine our current understanding of several areas of palliative care, with the ultimate message that palliative care is simply a novel term for the total care of a child and family, an approach that should be applied consistently and concurrently regardless of disease status. By improving familiarity with palliative care and building relationships with palliative care specialists, the paediatric oncology clinician will ensure that the best care possible for children and families is provided, regardless of outcome.

A sequential treatment algorithm for infants with stage 4s neuroblastoma and massive hepatomegaly

Infants with 4s neuroblastoma (NB) and massive hepatomegaly have a guarded prognosis and mortality approaches 30%. We report on eight patients with 4s NB and massive hepatomegaly treated with multiple modalities. One patient had spontaneous tumor regression. Three patients had progressive disease and responded to chemotherapy. Four patients progressed despite intravenous chemotherapy, of whom two died, and two were salvaged with hepatic intra‐arterial chemoembolization. Treatment of infants with stage 4s NB with massive hepatomegaly should be individualized based on disease course. A sequential approach with observation, intravenous chemotherapy, and intra‐arterial chemoembolization, may improve the outcome of these infants. Pediatr Blood Cancer 2012; 59: 182–184.

MSC for the improvement of hematopoietic engraftment.

A recent issue of Bone Marrow Transplantation included the results of a clinical trial from Minnesota of 15 pediatric patients who were transplanted with umbilical cord blood in combination with pre-expanded MSCs in an attempt to accelerate hematopoietic recovery.1 According to our present understanding, MSCs, osteoblasts and other stromal cells, including sinusoidal and endothelial, are responsible for supporting hematopoiesis and controlling HSC number. Therefore a co-transplantation of these cells, together with HSC, can be more effective in the reconstitution of hematopoiesis. This approach is especially attractive for cases with a predicted poor engraftment due to both an insufficient number of transplanted HSCs or impaired stroma.

Compassion fatigue, burnout and compassion satisfaction in neonatologists in the US.

Objective:Compassion fatigue (CF) is distress experienced by caregivers from ongoing contact with patients who are suffering. Burnout (BO) is occupational stress directly related to dissonance between job demands and available resources. Compassion satisfaction (CS) is professional fulfillment experienced through helping others. CF in physicians is not well studied. Neonatologists may be at particular risk for CF by virtue of recurrent exposure to distress in patients and their families. The objectives of this study were to determine the prevalence of CF, BO and CS, and to identify potential predictors for these phenomena in neonatologists.Study design:A modified Compassion Fatigue and Satisfaction Self-Test and a questionnaire of professional details and personal characteristics were distributed electronically to neonatologists nationally. Multivariable logistic and linear regression models for CF, BO and CS as a function of potential predictors were constructed.Results:The survey response rate was 47%. The prevalence of CF, BO and CS was 15.7, 20.8 and 21.9%, respectively. Female gender, emotional depletion, distress from ‘a clinical situation’, ‘co-workers’, ‘personal health issues’ and ‘not talking about distressing issues’ were each significant determinants of CF. Emotional depletion, distress from the ‘physical work environment’ and ‘co-workers’, and ‘not talking about distressing issues’ were significant determinants of BO. Self-identification as Hispanic; ‘not currently feeling distressed’; talking about distressing issues; and utilization of pediatric palliative care services were significant determinants of higher CS.Conclusions:CF and BO may impact emotional well-being and professional performance of neonatologists. Enhancement of CS is a potential target for intervention.

Spiritual Needs of Families With Bereavement and Loss of an Infant in the Neonatal Intensive Care Unit: A Qualitative Study

CONTEXT The hospital is a place full of distress and questions about the meaning of life. The death of a child can cause a spiritual struggle and crisis. Therefore, it is necessary for health care providers in the neonatal intensive care unit (NICU) to assess the spiritual needs of families that have lost a child. OBJECTIVES The purpose of this study was to explore the spiritual needs of families in Iran at the end of their babys life and through bereavement in the NICU. METHODS This study was an exploratory qualitative study performed using purposeful sampling and semi-structured interviews with 24 participants. Inclusion criteria for families, nurses, and physicians included having experienced at least one newborn death in the last six months in the NICU. The research environment was the NICU in Isfahan, one of the largest cities in Iran. RESULTS Data analysis revealed three main themes: spiritual belief in a supernatural power, the need for comfort of the soul, and human dignity for the newborn. CONCLUSION The results of this study created a new vision in addressing spiritual needs of Iranian families who experience the death of a newborn.

A rare case of GATA1 negative chemoresistant acute megakaryocytic leukemia in an 8‐month‐old infant with trisomy 21

Children with Down syndrome (DS) have a unique form of acute megakaryocytic leukemia (AMKL) characterized by the presence of mutations in the GATA1 gene leading to increased chemosensitivity and a favorable outcome. We describe an 8‐month‐old male with DS who was diagnosed with AMKL without a mutation in the GATA1 gene. The patient was treated according to the DS‐AML‐regimen but his disease progressed and he succumbed 9 months later. This rare case of DS AMKL without a GATA1 mutation with an unfavorable outcome suggests that GATA1 testing may play a useful role in initial stratification. Pediatr Blood Cancer 2010;54:1048–1049

Ewing Sarcoma: A 15-Year Experience of a Single Center With the MSKCC P6 Treatment Protocol.

Introduction: Ewing sarcoma (ES) is the second most common bone tumor in children. Current chemotherapeutic regimens include high-dose anthracyclines and alkylating agents with significant variation in treatment length. The Memorial Sloan Kettering Cancer Center P6 regimen (MSKCC P6) treatment protocol is a highly aggressive regimen given over 21 weeks only. We present the outcome of ES patients treated in our center with this protocol over the last 15 years. Procedure: We retrospectively analyzed data on the presentation, patient characteristics, treatment, and outcome of all ES patients treated according to the MSKCC P6 regimen from 1999 to 2014. Results: Of 48 patients, 37 (77%) presented with a nonmetastatic disease and 26 (54%) with tumor located in the extremities. The 5-year overall survival (OS) of the entire cohort was 55.9%±8%. Nonmetastatic disease conferred a better prognosis with a 5-year OS of 68.4%±8.5%. Patients with a nonmetastatic extremity tumor had the most favorable outcome with 5-year OS of 72.2%±9.8%. Conclusion: The outcome of ES patients after a short aggressive course of chemotherapy (the MSKCC P6 protocol), is comparable to that following other first-line treatment regimens in use, with potentially fewer long-term adverse events.

Isolated CNS vasculitis: Unusual presentation of relapsed Ewing sarcoma

We describe a 12‐year‐old boy male who presented with an expressive dysphasia after completion of treatment for unifocal Ewing sarcoma. CNS vasculitis was diagnosed by MRI/MRA and cerebral angiography. Extensive rheumatologic work‐up failed to identify an underlying primary process. Restaging studies showed no evidence of tumor. Complete neurologic recovery was achieved on prednisone. Four months later the patient developed overt, extensive metastases, confirmed by biopsy to represent recurrent Ewing sarcoma. Despite intensive therapy the patient succumbed 6 months later. This case demonstrates the unique finding of isolated CNS vasculitis as a presenting sign of Ewing sarcoma. Pediatr Blood Cancer 2010;54:326–328.

Charlie Gard: How Did Things Go Wrong?

Purpose of ReviewWe examine the discussions generated to date by the Charlie Gard case, as well as the events of the case itself, in order to examine lessons for providers dealing with similar situations in the future.Recent FindingsPublications regarding the Gard case are relatively few and focus primarily on the ethical and legal issues that arise when involving the court system in complex medical decision-making and potential limits to parental authority. Some publications have also addressed the subject of experimental therapies, especially from the perspective of potential harms, suffering, and cost.SummaryWe suggest early introduction of palliative care and careful attention to communication might reduce conflict and improve satisfaction for all involved parties. Likewise, we suggest limiting court system to truly extraordinary circumstances; all efforts should be made to avoid legal action and to honor and respect parental authority.

High symptom burden in children with cancer and high parental satisfaction: why the disconnect?

Pediatric palliative care (PC) has emerged over the past two decades as an approach aiming to ease suffering for children and their families coping with life-threatening illness, and has become increasingly integrated, concurrently, with disease-directed care. There is also recognition of the need for improved PC services in low- and middle-income countries. While most innovations occurring in PC are taking place in industrialized countries, over 90% of global child cancer deaths occur in low- and middle-income countries (1). Barriers to care include delayed diagnosis, limited access to therapies, insufficient and undertrained personnel and lack of infrastructure (2). Where services do exist, financial barriers may remain a significant concern (3). Especially frustrating is that access to even relatively inexpensive and easy to use treatments, such as morphine, remains inadequate due to both concrete and conceptual barriers (2,3).