Effectiveness of autologous hematopoietic stem cell transplantation in the treatment of high-risk neuroblastoma in children: a single-center clinical study / 中国当代儿科杂志

OBJECTIVES@#To investigate the effectiveness of high-dose chemotherapy combined with autologous hematopoietic stem cell transplantation (ASCT) in the treatment of children with high-risk neuroblastoma (NB).@*METHODS@#A retrospective analysis was performed on 29 children with high-risk NB who were admitted to Shanghai Children's Hospital and were treated with high-dose chemotherapy combined with ASCT from January 2013 to December 2021, and their clinical features and prognosis were analyzed.@*RESULTS@#Among the 29 children treated by high-dose chemotherapy combined with ASCT, there were 18 boys (62%) and 11 girls (38%), with a median age of onset of 36 (27, 59) months. According to the International Neuroblastoma Staging System, 6 children (21%) had stage III NB and 23 children (79%) had stage IV NB, and the common metastatic sites at initial diagnosis were bone in 22 children (76%), bone marrow in 21 children (72%), and intracalvarium in 4 children (14%). All 29 children achieved reconstruction of hematopoietic function after ASCT. After being followed up for a median time of 25 (17, 45) months, 21 children (72%) had continuous complete remission and 8 (28%) experienced recurrence. The 3-year overall survival rate and event-free survival rate were 68.9%±16.1% and 61.4%±14.4%, respectively. Presence of bone marrow metastasis, neuron-specific enolase ≥370 ng/mL and positive bone marrow immunophenotyping might reduce the 3-year event-free survival rate (P<0.05).@*CONCLUSIONS@#Children with high-risk NB who have bone marrow metastasis at initial diagnosis tend to have a poor prognosis. ASCT combined with high-dose chemotherapy can effectively improve the prognosis of children with NB with a favorable safety profile.

Aplicando la normativa protectora de los consumidores por deficiencias en advertencias de riesgo en el prospecto de medicamentos / Applying consumer protection rules for deficient risk warnings in medicine's package insert

El Tribunal de Apelaciones en lo Civil de 2º Turno, confirma la sentencia de primera instancia que, en aplicación de la normativa protectora de los consumidores en el ámbito del Derecho a la Salud, condenó al laboratorio farmacéutico fabricante de un medicamento con el que fue tratado un paciente para el acné, y que posteriormente desarrolló una aplasia medular. Aún sin quedar comprobada la causalidad entre la ingesta del medicamento y el daño sufrido por el paciente, la falta de advertencia en el prospecto de que se habían dado una serie de casos que se habrían asociado a la enfermedad aunque no comprobados, hace incurrir al fabricante en responsabilidad objetiva

The Court of Appeals of the second turn, confirmed the first instance sentence, which, through the application of the consumer protection rules to the field of Health Law, condemned a pharmaceutical company that manufactures a medicine used to treat a patient with acne who developed aplastic anemia. Despite the relationship of causality between the intake of the medicine and the damage suffered by the patient was not demonstrated, the lack of warning in the package insert about other cases, also not proven, that might be associated with this disease, causes the pharmaceutical laboratory incurs in objective responsibility.

Primary bone marrow B-cell non-hodgkin's lymphoma successfully treated with R-CHOP / Linfoma primario no hodgkin de células B en la médula ósea tratado con R-CHOP

Primary isolated bone marrow disease as a presenting feature of lymphoma is very rare. We describe the case of a Chinese with isolated bone marrow small B-cell lymphoma as a first manifestation. A 55-year old woman was admitted to our hospital with fever. Her peripheral blood smear and laboratory findings were suggestive of bicytopenia. Bone marrow specimen showed diffusely distributed small-sized lymphocytes. Combined with immunophenotypic and chromosomal analysis, a diagnosis of primary bone marrow B-cell non-Hodgkin's lymphoma was made. The patient was treated with R-CHOP (rituximab and cyclophosphamide, epirubicin, vindesine, and prednisone) regimen for six cycles. She had complete remission and is still alive without relapse. We concluded that primary bone marrow mature small B-cell lymphoma is a rare but distinctive subtype of lymphoma. The prognosis for this entity is poor but rituximab-based treatment is promising for improving its outcomes.

La enfermedad aislada primaria de la médula ósea como rasgo de manifestación del linfoma es muy rara. Describimos el caso de una paciente china con linfoma aislado de células B pequeñas en la médula como una primera manifestación. Se trata de una mujer de 55 años que ingresara a nuestro hospital con fiebre. El frotis de sangre periférica y los hallazgos de laboratorio apuntaban a una bicitopenia. El espécimen de la médula ósea mostró la presencia de linfocitos de pequeño tamaño distribuidos de manera difusa. En combinación con un análisis inmunofenotípico y un análisis cromosómico, se realizó un diagnóstico de linfoma primario no Hodgkin de células B de la médula ósea. La paciente recibió como tratamiento un régimen de seis ciclos de R-CHOP (rituximab, ciclofosfamida, epirubicina, vindesina, y prednisona). Esto le permitió alcanzar una remisión completa, y todavía está viva sin que se haya producido recaída alguna. Concluimos que el linfoma primario de células B pequeñas maduras de la médula ósea es un subtipo raro pero particular de linfoma. La prognosis para esta entidad es pobre, pero el tratamiento a base de rituximab re-basado resulta promisorio en cuanto a mejorar su evolución clínica.

Langerhans' cell histiocytosis [LCH]

Twelve patients with biopsy proven Langerhans cell histiocytosis [LCH] were diagnosed between 1983-1993 at King Fahd University Hospital, AI-Khobar, Saudi Arabia. There were eight males and four females with a ratio of 2: 1. Three has localized lesions and nine had disseminated disease with or without organ dysfunction. Bone involvement was seen in nine patients [75%] and five had organ dysfunction. Follow-up was from two to ten years M:5 yrs. Two patients died, three had recurrences and four [33%] had disabilities. Overall disease free survival was [83%]

Transplante de médula ósea de trilliza idéntica a paciente con hemoglobinuria paroxística nocturna (HPN) y anemia aplástica / Transplantation of bone marrow from triplet of patient with paroxysmal nocturnal hemoglobin (PNH) and aplastic anemia

Una paciente con hemoglobinuria paroxística nocturna y aplasia severa de la médula ósea fue tratada con transplante de la médula ósea de su hermana trilliza idéntica. La paciente fue preparada con ciclofosfamida y ha tenido recuperación medular completa y constante. Las pruebas que identifican la hemoglobinuria paroxística nocturna han sido persistentemente negativas. La paciente ha estado clínica y hematológicamente normal durante más de 8 años sin ninguna medicación