Lilian Piron-Ruiz

Crohn\'s disease patients effectively mobilize peripheral blood stem cells to perform autologous haematopoietic stem cell transplantation

Background Treatment with high doses chemotherapy followed by autologous haematopoietic stem cell transplantation is promising for refractory Crohn’s disease patients with no therapeutic option and at imminent risk of further surgeries. Objectives To evaluate the feasibility and efficacy of haematopoietic progenitor cell mobilization in a group of Crohn’s disease patients preparing for autologous unselected haematopoietic stem cell transplantation in a single institution. This is the first study to evaluate mobilization for Crohn’s disease. Methods Patients were selected according to criteria of the European Bone Marrow Transplant Society. Results All patients mobilized with the mean number of haematopoietic progenitor cells obtained and infused being 16.17 × 106/CD34+/kg. Most patients required only one leukapheresis session to reach the ideal number of cells. Grafting occurred around ten days after cells infusion. Complications and adverse events during the mobilization period were rare with only one patient presenting sepsis as a relevant event in the period. Most patients 20 (70%) had anaemia from the beginning of the mobilization but only 11 (37.9%) received packed red blood cell transfusions. Conclusion Mobilization in patients with Crohn’s disease is effective and it seems they are good mobilizers.

Hematopoietic stem cell transplantation for Crohn’s disease: Gaps, doubts and perspectives

Crohn’s disease (CD) is an inflammatory bowel disease that can affect any site of the digestive system. It occurs due to an immunological imbalance and is responsible for intestinal mucosal lesions and complications such as fistulas and stenoses. Treatment aims to stabilize the disease, reducing the symptoms and healing intestinal lesions. Surgical procedures are common in patients. Cell therapy was initially used to treat this disease in patients who also suffered from lymphoma and leukemia and were considered to be good candidates for autologous and allogeneic transplantation. After transplantation, an improvement was also observed in their CD. In 2003, the procedure began to be used to treat the disease itself, and several case series and randomized studies have been published since then; this approach currently comprises a new option in the treatment of CD. However, considerable doubt along with significant gaps in our knowledge continue to exist in relation to cell therapy for CD. Cell therapy is currently restricted to the autologous modality of hematopoietic stem cell transplantation and, experimentally, to mesenchymal stromal cells to directly treat lesions of the anal mucosa. This article presents the supporting claims for transplantation as well as aspects related to the mobilization regime, conditioning and perspectives of cell therapy.

Hematopoietic stem cell transplantation in a severe refractory Crohn’s disease patient with intestinal stoma: a case report

Background Hematopoietic stem cell transplantation (HSCT) can be used in the treatment of patients with refractory Crohn’s disease (CD) when no alternative treatment is available. However, HSCT increases the risk of infections, in particular during the aplasia of mobilization and conditioning. Moreover, intestinal stomas in CD augment the risk of morbidity in immunocompromised patients and under aplastic conditions. The objective of this report was to describe the results of the first year after HSCT in a CD patient with an intestinal stoma. Methods The patient was assessed in respect to disease symptoms and endoscopic findings before the procedure and 30, 90, 180, and 365 days after HSCT. Results No complications were observed during mobilization and conditioning with sufficient CD34+ cells being harvested in just one apheresis session. Toxicity was restricted to the hematological series. Scores of all the CD indexes and the quality of life of the patient improved. However, two of three endoscopic scores remained unchanged even though improvements were found in the appearance of the lesions. Conclusion HSCT may be an alternative treatment for refractory CD in patients with an intestinal stoma, and a priori, carefully selected patients with stomas should not be excluded as candidates for this procedure.

O G-CSF na terapia do acidente vascular cerebral

The granulocyte colony-stimulating-factor (G-CSF) is a glycoproteina which has been described for decades, and it is commonly utilized in the treatment of neutropenic states and bone marrow transplants. G-CSF stimulates hematopoietic stem-cels e crucially regulates the survival of mature neutrophils through a mechanism of apoptosis inhibition. Beyond its systemic effect, recently it has been shown its surprising activity in the central nervous system (CNS). G-CSF administration mobilizes bone marrow stem cells para systemic blood, and those cells cross the blood-brain-barrier e target brains damaged area. G-CSFs activity in the CNS has been defined as multimodal, because additionally it has been demonstrated a direct neuroprotective action through different mechanisms such as antiapoptotic activity, angiogenesis, anti-inflamatory effect, and stimulation of endogenous neurogenesis. This paper sumarizes G-CSF action in the CNS and approaches its potential para use in stroke.

O transplante de células-tronco hematopoéticas como opção no tratamento de doenças não hematológicas

Nesta revisao sao abordadas as doencas em que existem dados e perspectivas do uso de transplante de celulas-tronco hematopoeticas em suas diversas modalidades. Sao apresentados tambem os aspectos referentes aos regimes de condicionamento empregados, e sua relacao com toxicidade e taxa de mortalidade ligadas ao transplante. Sao apresentadas as doencas autoimunes e particularizados dados especificos do lupus eritematoso sistemico, esclerose sistemica e esclerose multipla e diabetes mellitus tipo 1. A base do procedimento nas doencas autoimunes e a reprogramacao imunologica. Aparentemente o procedimento tem sua indicacao nas doencas em que os tratamentos convencionais de imunossupressao tenham falhado, e o dano orgânico nao tenha sido definitivo, mas tenha chance de ocorrer caso nao seja realizado o transplante. A modalidade aparentemente indicada no momento deve ser o transplante de celulas-tronco autogenico com regimes de condicionamento nao mieloablativo para se obter sobrevivencia estimada em mais de 50% em todas as doencas, com baixa toxicidade e com mortalidade nula ligada ao transplante. Sao apresentados tambem os resultados nos tumores solidos, que sao discutiveis, e particularidades no câncer de mama. A aparente indicacao para os tumores solidos e transplante de celulas-tronco alogenico e se baseia no tratamento intensivo com doses mieloablativas com a finalidade de se induzir o efeito enxerto contra o tumor. Os regimes nao mieloablativos sao preconizados com a finalidade de reducao da toxicidade e inducao de imunossupressao, sendo os dados insuficientes e discutiveis, o que obriga a introducao de novas estrategias terapeuticas baseadas na terapia imune e celular.

Varicela hemorrágica após transplante de medula óssea

Apresentamos o caso de um paciente proceden-te de Goiânia, 27 anos, sexo masculino, portador deLeucemiaMieloideCronica,submetidoaTMO/MO,deirmao HLA identico, aos tres meses apos o diagnosticoemfasecronica,semincompatibilidadedogrupoABOeRH.Utilizoucomotratamentodesuadoencadebaseprevio ao TMO unicamente Hidroxiurea. Regime decondicionamento com busulfano 16 mg/kg eciclofosfamida 140 mg/kg, profilaxia de Doenca En-xerto Contra o Hospedeiro (DECH) com metotrexato16mg/m