AMOR: a proposed cooperative effort to improve outcomes of childhood cancer in Central America

The dramatic reduction of pediatric cancer mortality rates has been one of the greatest accomplishments of contemporary medicine. About 80% of children with cancer are now expected to be cured by current therapies. However, most of the world's children have no access to cancer treatment. The translation of effective pediatric cancer therapies to impoverished regions of the world presents an enormous challenge to the health care profession. Over the past 20 years, efforts have been under way to extend adequate cancer treatment to an increasing number of children in developing countries. These initiatives, collectively designated "twinning programs," consist essentially of a partnership between a pediatric cancer unit in a developing country and a group of health care providers in the developed world. Here we review the twinning programs that have been implemented in Central America, discuss their impact on the development of local resources and the outcome of childhood cancer, and propose a collaborative research initiative aimed at improving the international dissemination of progress in pediatric hematology-oncology.     

Establishment of a formal program for retinoblastoma: Feasibility of clinical coordination across borders and impact on outcome

Retinoblastoma is an ocular tumor that occurs in young children, in either heritable or sporadic manner. The relative rarity of retinoblastoma, and the need for expensive equipment, anesthesia, and pediatric ophthalmologic expertise, are barriers for effective treatment in developing countries. Also, with an average age‐adjusted incidence of two to five cases per million children, patient number limits development of local expertise in countries with small populations. Lebanon is a small country with a population of approximately 4.5 million. In 2012, a comprehensive retinoblastoma program was formalized at the Children's Cancer Institute (CCI) at the American University of Beirut Medical Center, and resources were allocated for efficient interdisciplinary coordination to attract patients from neighboring countries such as Syria and Iraq, where such specialized therapy is also lacking. Through this program, care was coordinated across hospitals and borders such that patients would receive scheduled chemotherapy at their institution, and monthly retinal examinations and focal laser therapy at the CCI in Lebanon. Our results show the feasibility of successful collaboration across borders, with excellent patient and physician adherence to treatment plans. This was accompanied by an increase in patient referrals, which enables continued expertise development. However, the majority of patients presented with advanced intraocular disease, necessitating enucleation in 90% of eyes in unilateral cases, and more than 50% of eyes in bilateral cases. Future efforts need to focus on expanding the program that reaches to additional hospitals in both countries, and promoting early diagnosis, for further improvement of globe salvage rates.     

SIOP PODC adapted treatment recommendations for standard‐risk medulloblastoma in low and middle income settings

Effective treatment of children with medulloblastoma requires a functioning multi‐disciplinary team with adequate neurosurgical, neuroradiological, pathological, radiotherapy and chemotherapy facilities and personnel. In addition the treating centre should have the capacity to effectively screen and manage any tumour and treatment‐associated complications. These requirements have made it difficult for many low and middle‐income countries (LMIC) centres to offer curative treatment. This article provides management recommendations for children with standard‐risk medulloblastoma (localised tumours in children over the age of 3–5 years) according to the level of facilities available. Pediatr Blood Cancer 2015;62:553–564. © 2014 Wiley Periodicals, Inc.     

Allogeneic CD34+ selected hematopoietic stem cell boost following CAR T-cell therapy in a patient with prolonged cytopenia and active infection

Hematological toxicity (hematotoxicity) leading to peripheral cytopenias is a common long-term adverse effect following the use of CD19-chimeric antigen receptor (CD19-CAR) T-cell therapies. However, management remains unclear for patients whose cytopenias persist beyond 1 month after CAR T-cell infusion. We present the case of a 21-year old who received CD19-CAR T-cell therapy for relapse following a haploidentical transplant. He developed hematotoxicity and consequently multiple life-threatening infections. We administered a CD34⁺ hematopoietic stem cell boost (HSCB) from his transplant donor, which led to hematopoietic recovery and resolution of his infections without any effect on the activity of CD19-CAR T cells. CD34⁺ HSCB can be a safe and effective option to treat hematotoxicity following CD19-CAR T-cell therapy.     

A case of luteinizing thecoma with sclerosing peritonitis: Revisiting a link with anti‐epileptic drugs

Luteinizing thecoma with sclerosing peritonitis (LTSP) is a rare ovarian tumor of unclear etiology and pathogenesis. The diagnostic entity was proposed in 1994, but a number of earlier reports described possible cases, and some suggested an association with anti‐epileptic drugs (AEDs). In presenting a new case we review the literature of previous cases to evaluate the possibility of such a link. When cases in reproductively immature patients are considered, evidence for an association between LTSP and AEDs is strongly suggested despite the rarity of the condition. Pediatr Blood Cancer 2010;54:470–472. © 2009 Wiley‐Liss, Inc.     

Romiplostim for therapy‐related thrombocytopenia in pediatric malignancies

Therapy‐related thrombocytopenia (TRT), due to chemotherapy and/or radiation therapy, is common with pediatric cancer treatments, and it can result in dose reductions and therapy delays. Romiplostim, a thrombopoietin mimetic, is efficacious as a second‐line treatment for immune thrombocytopenia in children and for TRT in adult cancer patients. However, there are no data for its use for TRT in children. We report a case series of five children treated for solid tumors where romiplostim was used without adverse effects to successfully resolve and prevent therapy‐limiting refractory TRT. Prospective studies on this use of romiplostim are warranted.