Rima Jubran

Langerhans Cell Histiocytosis (LCH): Guidelines for Diagnosis, Clinical Work-Up, and Treatment for Patients Till the Age of 18 Years

These guidelines for the management of patients up to 18 years with Langerhans cell histiocytosis (LCH) have been set up by a group of experts involved in the Euro Histio Net project who participated in national or international studies and in peer reviewed publications. Existing guidelines were reviewed and changed where new evidence was available in the literature up to 2012. Data and publications have been ranked according to evidence based medicine and when there was a lack of published data, consensus between experts was sought. Guidelines for diagnosis, initial clinical work‐up, and treatment and long‐term follow‐up of LCH patients are presented. Pediatr Blood Cancer 2013;60:175–184.

Predictors of outcome in children with Langerhans cell histiocytosis.

Our goal was to examine the clinical course of patients with Langerhans cell histiocytosis (LCH), with a special emphasis on bone disease and to attempt to identify and examine the factors that may predict reactivation and overall prognosis.

Approaches to treatment for extraocular retinoblastoma: Children's Hospital Los Angeles experience.

Extraocular retinoblastoma is associated with a very poor outcome. At Childrens Hospital Los Angeles, 10 of 207 patients with retinoblastoma had extraocular disease. Four patients with no histopathologic risk factors developed extraocular disease. All patients with direct extension into the central nervous system or with distant metastatic disease died. One of three patients with trilateral retinoblastoma and one patient with regional recurrence are alive after autologous bone marrow transplant. Patients with extraocular retinoblastoma who achieve remission may benefit from consolidation of their therapy with autologous bone marrow transplant.

Long-term outcomes of Group D eyes in bilateral retinoblastoma patients treated with chemoreduction and low-dose IMRT salvage.

To evaluate outcomes of Group D eyes of bilateral retinoblastoma patients treated with primary chemoreduction and external beam radiation as salvage.

High‐dose chemotherapy with autologous hematopoietic stem cell rescue for stage 4B retinoblastoma

Stage 4b retinoblastoma (central nervous system metastatic disease) has been lethal in virtually all cases reported. Here we describe a series of eight patients treated with intensive chemotherapy, defined as the intention to include high‐dose chemotherapy with autologous hematopoietic stem cell rescue.

Trilateral retinoblastoma: potentially curable with intensive chemotherapy.

Trilateral retinoblastoma has been lethal in virtually all cases previously reported. We describe a series of 13 patients treated with intensive chemotherapy, defined as the intention to include high‐dose chemotherapy with autologous hematopoietic stem cell rescue.

Choroid plexus tumors; management, outcome, and association with the Li–Fraumeni syndrome: The Children's Hospital Los Angeles (CHLA) experience, 1991–2010†

Choroid plexus tumors (CPT) are rare, and predominate in early childhood. An association with the Li–Fraumeni syndrome (LFS) has been reported, but the biological and clinical implications of this association remain poorly defined. We have investigated the clinical features and overall survival of all CPT patients treated at Childrens Hospital Los Angeles (CHLA) over a 20‐year period, with particular attention to the association of CPT with LFS.

High-dose chemotherapy and autologous hematopoietic progenitor cell rescue in children with recurrent medulloblastoma and supratentorial primitive neuroectodermal tumors: the impact of prior radiotherapy on outcome.

The role of myeloablative chemotherapy in children with recurrent medulloblastoma and supratentorial primitive neuroectodermal tumors (MB/ST‐PNET) is controversial, in particular in patients who develop recurrent disease after craniospinal radiotherapy.

Brainstem primitive neuroectodermal tumors (bstPNET): results of treatment with intensive induction chemotherapy followed by consolidative chemotherapy with autologous hematopoietic cell rescue.

We have evaluated the response rate and survival utilizing intensified chemotherapy followed by myeloablative chemotherapy with autologous hematopoietic cell rescue (AuHCR) and adjuvant radiation therapy in six young children with newly diagnosed brainstem primitive neuroectodermal tumors (bstPNET). Following maximum surgical resection of the tumor, patients received high dose induction chemotherapy including vincristine, cisplatin, cyclophosphamide, and etoposide. Eligible patients received a single cycle of myeloablative chemotherapy followed by AuHCR. Two patients survive at least 32 months with stable disease. This approach provides an alternative for young patients with bstPNET who in prior reports have had a uniformly fatal prognosis. Pediatr Blood Cancer 2008;50:715–717.

Management of retinoblastoma with proximal optic nerve enhancement on MRI at diagnosis.

In North America, retinoblastoma rarely presents with gross clinical evidence of tumor involving the optic nerve. Extent of microscopic tumor infiltration into the postlaminar optic nerve is a significant risk factor for metastasis, especially if there is tumor at the cut end. Due to poor outcomes in patients with metastatic disease, historical treatment for patients with clinical evidence of extraocular optic nerve involvement has included upfront enucleation followed by aggressive adjuvant chemotherapy. Additional orbital irradiation is advocated for individuals with optic nerve involvement at the surgical margin. Little is known about the role of neoadjuvant therapy in the setting of orbital optic nerve enhancement on magnetic resonance imaging (MRI) at diagnosis.