Your browser doesn't support javascript.
Mostrar: 20 | 50 | 100
Resultados 1 - 20 de 22
Filtrar
Mais filtros










Base de dados
Intervalo de ano de publicação
1.
J Clin Apher ; 34(3): 171-354, 2019 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-31180581

RESUMO

The American Society for Apheresis (ASFA) Journal of Clinical Apheresis (JCA) Special Issue Writing Committee is charged with reviewing, updating and categorizing indications for the evidence-based use of therapeutic apheresis (TA) in human disease. Since the 2007 JCA Special Issue (Fourth Edition), the committee has incorporated systematic review and evidence-based approaches in the grading and categorization of apheresis indications. This Eighth Edition of the JCA Special Issue continues to maintain this methodology and rigor in order to make recommendations on the use of apheresis in a wide variety of diseases/conditions. The JCA Eighth Edition, like its predecessor, continues to apply the category and grading system definitions in fact sheets. The general layout and concept of a fact sheet that was introduced in the Fourth Edition, has largely been maintained in this edition. Each fact sheet succinctly summarizes the evidence for the use of TA in a specific disease entity or medical condition. The Eighth Edition comprises 84 fact sheets for relevant diseases and medical conditions, with 157 graded and categorized indications and/or TA modalities. The Eighth Edition of the JCA Special Issue seeks to continue to serve as a key resource that guides the utilization of TA in the treatment of human disease.


Assuntos
Remoção de Componentes Sanguíneos/métodos , Medicina Baseada em Evidências/normas , Humanos , Terapêutica/métodos , Estados Unidos , Redação
2.
J Clin Apher ; 33(4): 464-468, 2018 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-29575114

RESUMO

Stimulated by the scientific progress in deciphering the principal elements contributing to the clinical efficacy of extracorporeal photochemotherapy (ECP), the American Council on ECP (ACE) was formed, under the auspices of the American Society for Apheresis (ASFA), to develop a field-guiding Consensus Report. ACE is composed of thirty nationally recognized ECP experts, clinically spanning cancer, transplantation, and autoimmunity and scientifically bridging immunology, bioengineering, and hematology. The two-day meeting took place in Manhattan, April 13-14, 2017, and unanimous consensus on nine pivotal points is herein reported. (1) ECP's clinical evolution must now enter a scientifically driven phase. (2) ECP is currently a bidirectional therapy, both immunizing and tolerizing simultaneously, via a single one-size-fits-all inflexible medical device. (3) To preclude inadvertent tolerization in the cancer setting, or immunization in the transplant rejection setting, polarization of ECP to either immunization or tolerization mode to match the clinical need is now possible and necessary. (4) Cutaneous T cell lymphoma (CTCL) is a genetically driven cancer, whose response to ECP is due to enhanced anti-cancer immunity. (5) ECP is a dendritic antigen-presenting cell (DC) based therapy. (6) ECP's efficacy can now be tested in a broad array of cancers. (7) ECP's capacity to tolerize to allotransplants via processing of donor leukocytes merits expedited human investigation. (8) UVA-8-MOP-impacted ECP-induced DC are potent antigen-specific tolerizing agents, while UVA-8-MOP(8-Methoxypsoralen)-spared ECP-induced DC are potent antigen-specific immunizing agents. (9) Six pilot clinical trial areas (CTCL, graft-vs.-host disease, ovarian carcinoma, anti-graft cytotoxic antibodies, pemphigus vulgaris, and haplotype mismatched stem cell transplants) are advised. ACE will be an ongoing advisory group for the field, with the goal of overseeing coordinated clinical and fundamental research efforts.


Assuntos
Fotoferese/métodos , Animais , Conferências de Consenso como Assunto , Células Dendríticas/imunologia , Rejeição de Enxerto/terapia , Humanos , Neoplasias/terapia
3.
Hematol Oncol Stem Cell Ther ; 11(3): 169-174, 2018 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-29397331

RESUMO

BACKGROUND: Acute graft versus host disease (aGVHD) affects approximately 30-60% of patients after allogeneic hematopoietic stem cell transplantation (HCT) and our ability to predict who develops this complication and their response to treatment is limited. Fecal calprotectin has recently gained popularity as an effective marker of GI inflammation in patients with Inflammatory Bowel Disease (IBD). METHODS: Fecal calprotectin and albumin were evaluated as prognostic and predictive markers of aGVHD in 60 adult and pediatric HCT patients. Stool samples were sent for calprotectin quantification prior to starting conditioning, at day 14 post-HCT, at day 28 post-HCT, and at onset of aGVHD ±â€¯2 days. RESULTS: Fecal calprotectin did not differentiate patients with GI-GVHD and non-GI GVHD and did not vary based on severity. However, in patients with steroid-refractory GI aGVHD, significantly higher fecal calprotectin levels were noted. At onset of lower-GI symptoms, steroid refractory patients (n = 3) had a mean fecal calprotectin level of 449 ug/g (range 116-1111 ug/g) and a mean albumin of 1.93 g/dL (range 1.6-2.3 g/dL) compared with a mean fecal calprotectin of 24 ug/g (range 16-31 ug/g) and a mean albumin of 3.3 g/dL (range 2.3-3.9 g/dL) in steroid responsive patients (n = 9) (fecal calprotectin p = 0.032, albumin p = 0.027). CONCLUSION: Patients with steroid-refractory GI aGVHD had higher fecal calprotectin levels and lower albumin levels than patients with steroid-responsive disease. We recommend further studies to evaluate non-invasive tests with fecal calprotectin in combination with albumin in predicting steroid refractory disease at onset of symptoms to potentially identify patients that may benefit from upfront escalation in GVHD treatment.


Assuntos
Doença Enxerto-Hospedeiro/metabolismo , Transplante de Células-Tronco Hematopoéticas , Doenças Inflamatórias Intestinais/metabolismo , Complexo Antígeno L1 Leucocitário/metabolismo , Albumina Sérica/metabolismo , Adolescente , Adulto , Idoso , Aloenxertos , Biomarcadores/metabolismo , Criança , Pré-Escolar , Anemia de Fanconi/metabolismo , Anemia de Fanconi/terapia , Fezes , Feminino , Doença Enxerto-Hospedeiro/etiologia , Neoplasias Hematológicas/metabolismo , Neoplasias Hematológicas/terapia , Humanos , Doenças Inflamatórias Intestinais/etiologia , Masculino , Pessoa de Meia-Idade
4.
J Pediatr Hematol Oncol ; 40(1): 31-35, 2018 01.
Artigo em Inglês | MEDLINE | ID: mdl-28538090

RESUMO

Multimodal treatment in high-risk neuroblastoma has modestly improved survival; limited data exist on the late effects from these regimens. We report the sequelae of treatment incorporating 3 consecutive cycles of high-dose therapy and autologous stem cell transplants (ASCTs) without the use of total body irradiation (TBI). We reviewed the medical records of 61 patients treated on or following the Chicago Pilot 2 protocol between 1991 and 2008. Of the 25 patients who are alive (41%), 19 had near complete data to report. Specific treatment modalities and therapy-related side effects were collected. Fourteen of these 19 patients (74%) received 3 cycles of high-dose therapy with ASCT; follow-up occurred over a median of 13.9 years (range, 5.8 to 18.8 y). The majority of late effects were endocrine-related, including growth failure, hypothyroidism, and hypogonadism. Patients also developed secondary neoplasms and skeletal deformities. The most frequent sequela was hearing loss, seen in 17/19 patients. We found a high prevalence of various late effects in survivors of high-risk neuroblastoma using a non-TBI-based regimen including 3 cycles of high-dose therapy with ASCTs. As current treatment regimens recommend tandem ASCT without TBI, it is imperative that we understand and monitor for the sequelae from these modalities.


Assuntos
Quimioterapia de Consolidação/métodos , Transplante de Células-Tronco Hematopoéticas/métodos , Quimioterapia de Indução/métodos , Neuroblastoma/terapia , Sobreviventes , Pré-Escolar , Terapia Combinada/métodos , Terapia Combinada/mortalidade , Quimioterapia de Consolidação/efeitos adversos , Feminino , Seguimentos , Transplante de Células-Tronco Hematopoéticas/mortalidade , Humanos , Quimioterapia de Indução/efeitos adversos , Lactente , Masculino , Agonistas Mieloablativos , Neuroblastoma/complicações , Neuroblastoma/mortalidade , Análise de Sobrevida , Transplante Autólogo
5.
Pediatr Hematol Oncol ; 35(5-6): 316-321, 2018.
Artigo em Inglês | MEDLINE | ID: mdl-30681039

RESUMO

Wilms tumor (WT) treatment regimens are curative for more than 80% of patients, but those with relapsed or refractory disease continue to have poor outcomes. High-dose chemotherapy followed by autologous stem cell rescue is often utilized although outcomes remain variable. We report on HD-ASCR outcomes in 24 patients with relapsed or refractory Wilms tumor. Three-year disease free and overall survival are 46% and 60%, respectively, which is similar to those reported for conventional salvage therapies. These outcomes suggest that conventional salvage therapies should be employed for relapsed and refractory WT rather than HD-ASCR.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Transplante de Células-Tronco Hematopoéticas , Neoplasias Renais , Recidiva Local de Neoplasia , Terapia de Salvação , Tumor de Wilms , Adolescente , Autoenxertos , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Seguimentos , Humanos , Neoplasias Renais/mortalidade , Neoplasias Renais/terapia , Masculino , Recidiva Local de Neoplasia/mortalidade , Recidiva Local de Neoplasia/terapia , Estudos Retrospectivos , Taxa de Sobrevida , Tumor de Wilms/mortalidade , Tumor de Wilms/terapia
6.
Hematology Am Soc Hematol Educ Program ; 2017(1): 639-644, 2017 12 08.
Artigo em Inglês | MEDLINE | ID: mdl-29222315

RESUMO

Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative option for many disease states. Despite significant improvements in strategies used to prevent and treat acute and chronic graft-versus-host disease (a/cGVHD), they continue to negatively affect outcomes of HSCT significantly. Standard, first-line treatment consists of corticosteroids; beyond this, there is little consistency in therapeutic regimens. Current options include the addition of various immunosuppressive agents, the use of which puts patients at even higher risks for infection and other morbidities. Extracorporeal photopheresis (ECP) is a widely used cellular therapy currently approved by the US Food and Drug Administration for use in patients with cutaneous T-cell lymphoma; it involves the removal of peripherally circulating white blood cells, addition of a light sensitizer, exposure to UV light, and return of the cells to the patient. This results in a series of events ultimately culminating in transition from an inflammatory state to that of tolerance, without global immunosuppression or known long-term adverse effects. Large-scale, prospective studies of the use of ECP in patients with a/cGVHD are necessary in order to develop the optimal treatment regimens.


Assuntos
Doença Enxerto-Hospedeiro/terapia , Fotoferese/métodos , Doença Aguda , Aloenxertos , Doença Crônica , Doença Enxerto-Hospedeiro/sangue , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas , Humanos , Linfoma Cutâneo de Células T/terapia
7.
J Clin Apher ; 32(6): 543-552, 2017 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-28304115

RESUMO

In adults, extracorporeal photopheresis (ECP) is widely utilized for a variety of indications, most commonly cutaneous T-cell lymphoma, acute or chronic graft-versus-host disease (GVHD), solid organ transplant rejection, and other autoimmune and T-cell-mediated disorders. In pediatric patients, the majority of case series and reports have focused on its use in the management of acute and chronic GVHD. Currently utilized ECP technologies were designed for adult patients and there are several challenges in adapting these technologies for use in children. In our review, we focus on practical considerations and procedural modifications for ECP use in pediatric patients, with special attention to patient safety.


Assuntos
Fotoferese/métodos , Criança , Doença Enxerto-Hospedeiro/terapia , Humanos , Pediatria/métodos , Fotoferese/normas , Avaliação da Tecnologia Biomédica
8.
Pediatr Blood Cancer ; 64(9)2017 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-28266766

RESUMO

Patients with acute myeloid leukemia (AML) who relapse after hematopoietic stem cell transplantation (HCT) have dismal outcomes. Our ability to predict those at risk for relapse is limited. We examined chimerism trends post-HCT in 63 children who underwent HCT for AML or myelodysplastic syndrome (MDS). Mixed T-cell chimerism at engraftment and absence of chronic graft versus host disease (cGVHD) were associated with relapse (P = 0.04 and P = 0.02, respectively). Mixed T-cell chimerism at engraftment was predictive in patients without cGVHD (P = 0.03). Patients with engraftment mixed T-cell chimerism may warrant closer disease monitoring and consideration for early intervention.


Assuntos
Leucemia Mieloide Aguda/imunologia , Leucemia Mieloide Aguda/terapia , Síndromes Mielodisplásicas/imunologia , Síndromes Mielodisplásicas/terapia , Linfócitos T/imunologia , Adolescente , Criança , Pré-Escolar , Quimerismo , Feminino , Transplante de Células-Tronco Hematopoéticas , Humanos , Lactente , Masculino , Recidiva Local de Neoplasia , Estudos Retrospectivos , Quimeras de Transplante , Adulto Jovem
9.
J Pediatr Hematol Oncol ; 39(4): 282-286, 2017 05.
Artigo em Inglês | MEDLINE | ID: mdl-28060108

RESUMO

Thrombotic complications are a significant source of morbidity and mortality following hematopoietic stem cell transplants. Among them, transplant-associated thrombotic microangiopathy (TA-TMA) is a well-recognized syndrome that can affect various organ systems. Its etiology is related to endothelial injury accompanied by complement activation. As many of the signs and symptoms of the disease are also encountered in other complications following hematopoietic stem cell transplant, it can often be difficult to establish the diagnosis based on clinical data alone. Histopathologic examination of various tissues may be performed in difficult cases. However, the microscopic features of TA-TMA also overlap with those seen in other posttransplant complications, suggesting a need for additional tests to help in diagnosis. Here we describe a patient who presented with hemolytic anemia, thrombocytopenia, renal and neurological impairment, who also developed significant bloody diarrhea. Flexible sigmoidoscopy with biopsies was performed to determine the exact etiology of his gastrointestinal bleed. A diagnosis of intestinal TA-TMA was established with the use of immunohistochemical stains for complement components C5b-9 and C4d. This is the first report that highlights the utility of complement staining on histologic sections from digestive samples to render a definitive diagnosis of intestinal TA-TMA.


Assuntos
Enteropatias/diagnóstico , Transplante de Células-Tronco/efeitos adversos , Microangiopatias Trombóticas/diagnóstico , Criança , Complemento C4b/análise , Complemento C5b/análise , Hemorragia Gastrointestinal/etiologia , Humanos , Imuno-Histoquímica , Masculino , Fragmentos de Peptídeos/análise , Microangiopatias Trombóticas/patologia
10.
J Clin Apher ; 32(4): 215-223, 2017 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-27442906

RESUMO

BACKGROUND: Although many apheresis centers offer extracorporeal photopheresis (ECP), little is known about current treatment practices. METHODS: An electronic survey was distributed to assess ECP practice internationally. RESULTS: Of 251 responses, 137 met criteria for analysis. Most respondents were from North America (80%). Nurses perform ECP at most centers (84%) and the majority of centers treat adults only (52%). Most centers treat fewer than 50 patients/year (83%) and perform fewer than 300 procedures/year (70%). Closed system devices (XTS and/or Cellex) are used to perform ECP at most centers (96%). The most common indications for ECP are acute/chronic skin graft versus host disease (89%) and cutaneous T-cell lymphoma (63%). The typical wait time for ECP treatment is less than 2 weeks (91%). Most centers do not routinely perform quality control assessment of the collected product (66%). There are device-specific differences in treatment parameters. For example, XTS users more frequently have a minimum weight limit (P = 0.003) and use laboratory parameters to determine eligibility for treatment (P = 0.03). Regardless of device used, the majority of centers assess the clinical status of the patient before each procedure. Greater than 50% of respondents would defer treatment for hemodynamic instability due to active sepsis or heart failure, positive blood culture in the past 24 h or current fever. CONCLUSION: This survey based study describes current ECP practices. Further research to provide evidence for optimal standardization of patient qualifications, procedure parameters and product quality assessment is recommended.


Assuntos
Fotoferese/métodos , Padrões de Prática Médica/normas , Doença Enxerto-Hospedeiro/terapia , Humanos , Linfoma Cutâneo de Células T/terapia , Seleção de Pacientes , Padrões de Prática Médica/tendências , Garantia da Qualidade dos Cuidados de Saúde , Transplante de Pele/efeitos adversos , Inquéritos e Questionários
11.
J Pediatr Hematol Oncol ; 39(1): 26-32, 2017 01.
Artigo em Inglês | MEDLINE | ID: mdl-27820121

RESUMO

Current practice for selecting donor units for umbilical cord blood transplant (UCBT) involves matching at HLA-A and HLA-B by low-resolution typing and the HLA-DRB1 allele by high-resolution (HR) typing. We retrospectively studied the impact of HR allele matching at HLA-A, HLA-B, HLA-C, and HLA-DRB1 on transplant-related outcomes in 60 single-unit UCBTs in pediatric patients with malignant and nonmalignant conditions. Five-year overall survival of our cohort was 71% (95% confidence interval, 58-81); 27% experienced primary graft failure. Applying HR typing, donor-recipient mismatch variability increased ranging from 1/8 to 8/8, however, no impact on primary graft failure, graft-versus-host disease or posttransplant infection was observed. UCBTs with ≥6/8 HR matches did have a better overall survival (P=0.04) and decreased transplant-related mortality (P=0.02) compared with <6/8 HR matches. Using standard HLA typing, we showed an increased incidence of acute graft-versus-host disease (grade II to IV) and decreased transplant-related mortality in comparing the matched (6/6) versus ≤5/6 group (P=0.05 and 0.05, respectively). These data support the use of current guidelines for umbilical cord blood selection and encourage utilization of HR typing to select umbilical cord blood units matched at ≥6/8 especially when appropriate ≥5/6 units are available.


Assuntos
Transplante de Células-Tronco de Sangue do Cordão Umbilical , Antígenos HLA/análise , Cadeias HLA-DRB1/análise , Teste de Histocompatibilidade/métodos , Doença Aguda , Adolescente , Alelos , Criança , Pré-Escolar , Transplante de Células-Tronco de Sangue do Cordão Umbilical/efeitos adversos , Transplante de Células-Tronco de Sangue do Cordão Umbilical/mortalidade , Feminino , Genes MHC Classe I , Genes MHC da Classe II , Doenças Genéticas Inatas/terapia , Sobrevivência de Enxerto , Doença Enxerto-Hospedeiro/epidemiologia , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/prevenção & controle , Antígenos HLA/genética , Cadeias HLA-DRB1/genética , Doenças Hematológicas/terapia , Humanos , Lactente , /etiologia , Isoanticorpos/biossíntese , Estimativa de Kaplan-Meier , Masculino , Neoplasias/mortalidade , Neoplasias/terapia , Disfunção Primária do Enxerto/epidemiologia , Disfunção Primária do Enxerto/etiologia , Disfunção Primária do Enxerto/prevenção & controle , Recidiva , Estudos Retrospectivos , Resultado do Tratamento , Ativação Viral
12.
J Clin Apher ; 31(1): 11-5, 2016 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-26275240

RESUMO

PURPOSE: Wilson's disease is a rare autosomal recessive genetic disorder that results in accumulation of copper in the liver, brain, cornea and kidney. Therapeutic plasma exchange (TPE) has been used to remove copper and provide a bridge to liver transplantation. We report here the collective experiences through the ASFA apheresis registry on Wilson's disease. METHODS: The ASFA apheresis registry is a multi-center registry study. Both prospective and retrospective data, with the latter involving data collection back to January 2000 are entered in the registry. The registry includes patient demographics, apheresis procedural information, treatment schedules, and treatment outcomes and complications. RESULTS: A total of 10 patients (3 males and 7 females) with Wilson's disease treated between 2005 and 2013 were included. Median age of first diagnosis and first TPE were 16 and 17 years, respectively. Via central venous access, these patients underwent a total of 43 TPEs; the median number of TPE procedures per patient was 3.5. All of the TPEs used ACD-A as anticoagulation, 42/43 TPEs targeted 1-1.25 plasma volumes, and 41/43 TPEs were performed with 100% fluid balance. Post TPE procedures, 9 patients underwent liver transplantation; all 10 patients had at least a 6-month survival. CONCLUSIONS: All 10 patients with Wilson's disease who underwent TPE had a positive outcome in terms of 6-month survival. In this first report of the ASFA apheresis registry study, we have demonstrated the value of using this registry to collect apheresis-related patient outcomes from multiple centers.


Assuntos
Remoção de Componentes Sanguíneos , Degeneração Hepatolenticular/terapia , Adolescente , Adulto , Criança , Feminino , Degeneração Hepatolenticular/complicações , Humanos , Falência Hepática Aguda/etiologia , Falência Hepática Aguda/terapia , Transplante de Fígado , Masculino , Pessoa de Meia-Idade , Troca Plasmática , Estudos Prospectivos , Sistema de Registros , Estudos Retrospectivos , Resultado do Tratamento , Adulto Jovem
13.
Biol Blood Marrow Transplant ; 21(9): 1612-21, 2015 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-26025482

RESUMO

Busulfan (Bu) is widely used in conditioning regimens for infants undergoing allogeneic hematopoietic progenitor cell transplantation (HPCT), but the best approach to administer Bu in this population is still unknown. Here, we report a single-center experience of the use of a test dose to guide dose adjustment of intravenous (i.v.) Bu therapy in infants. Between 2004 and 2013, 33 infants younger than 1 year with nonmalignant conditions received allogeneic peripheral blood or cord blood HPCT after a reduced-intensity conditioning (RIC) regimen consisting of fludarabine, antithymocyte globulin, and 2 single daily doses of i.v. Bu. Pharmacokinetic results of a test dose of i.v. Bu (.8 mg/kg) were used to determine the dose of 2 single daily i.v. Bu regimen doses, adjusted to target an area under the curve (AUC) of 4000 µMol*minute per day in a first cohort (n = 12) and 5000 µMol*minute in a second cohort (n = 21). The mean Bu clearance in our infant patients was found to be 3.67 ± 1.03 mL/minute/kg, and the test dose clearance was highly predictive of the regimen dose clearance. The mean AUC achieved after the first single daily regimen dose was 3951 ± 1239 in the AUC 4000 cohort and 4884 ± 766 for the AUC 5000 cohort. No patient in either cohort developed hepatic sinusoidal obstructive syndrome or seizures attributable to Bu. Primary graft failure occurred in 4 patients and secondary graft failure occurred in 3, predominantly in the AUC 4000 cohort (6 of 7). Among the engrafted patients (n = 28), 16 achieved full donor chimerism and 9 patients attained stable mixed chimerism. Overall survival of patients at 6 years after transplantation was 59.5% for the AUC 4000 cohort and 85.4% for the AUC 5000 cohort, with primary graft failure in the first cohort being a major contributor to morbidity. Logistic regression analysis showed that the risk of graft failure increased significantly if cord blood hematopoietic progenitor cells were used or if total Bu exposure was below 4000 µMol*minute per day for 2 days. The difference in clinical outcomes between the 2 cohorts supports the conclusion that targeting a higher Bu AUC of 5000 µMol*minute per day for 2 days improves donor engraftment in infants with nonmalignant conditions undergoing RIC HPCT without increasing toxicity. Measuring i.v. Bu pharmokinetics using a test dose allows timely adjustment of single daily regimen doses and optimization of total Bu exposure, resulting in an effective and safe regimen for these infants.


Assuntos
Bussulfano/administração & dosagem , Transplante de Células-Tronco de Sangue do Cordão Umbilical , Transplante de Células-Tronco Hematopoéticas , Condicionamento Pré-Transplante , Aloenxertos , Bussulfano/efeitos adversos , Intervalo Livre de Doença , Feminino , Seguimentos , Humanos , Lactente , Recém-Nascido , Masculino , Taxa de Sobrevida
14.
Platelets ; 26(7): 702-4, 2015.
Artigo em Inglês | MEDLINE | ID: mdl-25548835

RESUMO

Glanzmann thrombasthenia (GT) is a rare, autosomal recessive coagulopathy characterized by either qualitative or quantitative abnormalities of the membrane glycoprotein αIIbß3 complex leading to bleeding tendencies, ranging from purpura to life-threatening hemorrhage. Although patients can be managed with supportive measures including platelet transfusions, complications such as alloimmunization are possible. Allogeneic stem cell transplantation (ASCT) can be indicated in severe cases of GT. We report the case of an eight-month-old girl diagnosed with moderate-severe GT, who was successfully treated with a reduced-intensity, human leukocyte antigen (HLA)-identical ASCT.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Trombastenia/terapia , Plaquetas/imunologia , Plaquetas/metabolismo , Feminino , Antígenos HLA/genética , Antígenos HLA/imunologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/métodos , Teste de Histocompatibilidade , Humanos , Lactente , Índice de Gravidade de Doença , Irmãos , Trombastenia/diagnóstico , Condicionamento Pré-Transplante , Transplante Homólogo , Resultado do Tratamento
15.
Transfus Med Rev ; 29(1): 62-70, 2015 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-25459074

RESUMO

The clinical use of extracorporeal photopheresis (ECP) for accepted indications such as graft-versus-host disease, transplant rejection, and cutaneous T-cell lymphoma continues to increase. Expanded applications for ECP, such as the treatment of select autoimmune diseases, are being explored. Extracorporeal photopheresis's capacity to both immunotolerize in the autoreactive setting, while immunizing against a lymphoma is unusual and suggestive of a unique mechanism. It is likely that ECP's induction of dendritic cells is key to its efficacy in both of these settings, but exactly how ECP impacts other immune components and their interactions is not fully understood. Further basic science research is necessary to elucidate how these dissimilar cellular activities are functionally integrated. On the clinical side, collaborative multicenter trials designed to recognize the principal variables controlling therapeutic responses and improve prognostic indicators may enable tailoring devices, treatment schedules, and doses to the needs of the individual patients or diseases. This review describes our current understanding of how ECP influences the immune system, reviews the existing clinical applications of ECP, and explores areas for future basic science and clinical research as presented at the National Institutes of Health State of the Science Symposium in Therapeutic Apheresis in November 2012.


Assuntos
Pesquisa Biomédica , Remoção de Componentes Sanguíneos/tendências , Congressos como Assunto , Fotoferese , Pesquisa Biomédica/métodos , Pesquisa Biomédica/tendências , Remoção de Componentes Sanguíneos/métodos , Remoção de Componentes Sanguíneos/estatística & dados numéricos , Congressos como Assunto/organização & administração , Células Dendríticas/citologia , Humanos , Células Matadoras Naturais/citologia , National Institutes of Health (U.S.)/organização & administração , Fotoferese/estatística & dados numéricos , Linfócitos T/citologia , Estados Unidos
16.
Pediatr Blood Cancer ; 61(8): 1350-6, 2014 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-24634399

RESUMO

BACKGROUND: Outcomes for high-risk neuroblastoma remain poor. Modern treatment protocols utilizing intense induction followed by myeloablative consolidation chemotherapy with autologous stem cell rescue (ASCR) have improved survival rates, but the long-term sequelae, including development of secondary malignant neoplasms (SMN), are just now surfacing. METHODS: We retrospectively reviewed data from 87 patients with high-risk neuroblastoma who were treated with intensive induction chemotherapy followed by ASCR between January 1991 and July 2011 following one of two institutional protocols: Chicago Pilot 1 (CP1; n = 12) and Chicago Pilot 2 (CP2; n = 75). RESULTS: The 15-year overall survival rate for all 87 patients was 33.9% (95% confidence interval [CI], 23.1-45.0%). The 10- and 15-year cumulative incidence of SMN was 16.5% (95%CI, 7.2-38.0%) and 34.2% (95%CI, 18.6-63.1%), respectively, without evidence of a plateau at 15 years. Six of the 10 patients (n = 2 in CP1 and n = 8 in CP2) who developed SMN had hematologic malignancies including acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS). Solid tumors included thyroid papillary carcinoma, chondrosarcoma, hepatocellular carcinoma, and biliary adenocarcinoma. CONCLUSION: A significantly higher incidence of SMN, especially hematological malignancies, was observed in this cohort compared to older neuroblastoma studies, potentially due to exposure to epipodophyllotoxins and a high cumulative dose of alkylating agents these patients received. The risk of developing an SMN continued to increase with survival time and did not reach the plateau at 15 years. Although the number of the patients is relatively small, our study emphasizes the need for life-long follow-up of survivors who were treated using modern therapy.


Assuntos
Segunda Neoplasia Primária , Neuroblastoma , Transplante de Células-Tronco , Adolescente , Autoenxertos , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Seguimentos , Humanos , Incidência , Lactente , Masculino , Segunda Neoplasia Primária/mortalidade , Segunda Neoplasia Primária/terapia , Neuroblastoma/mortalidade , Neuroblastoma/terapia , Estudos Retrospectivos , Fatores de Risco , Taxa de Sobrevida
17.
J Clin Apher ; 29(2): 120-6, 2014 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-24105856

RESUMO

INTRODUCTION: Pediatric apheresis (PA) has distinct characteristics compared to adult apheresis, and requires specialized knowledge and experience to perform safely, particularly in low-weight patients. As evidence-based medicine advances the field of therapeutic apheresis, increased attention must be paid to pediatric patients with conditions for which apheresis is indicated. METHODS: An electronic survey of >5,000 potential participants throughout the world was conducted to ascertain the scope and the current state of practice. RESULTS: The survey elicited 159 responses from 12 countries; 107 of the responses provided sufficient information for analysis. Participants performed an average of 176 PA procedures/year (range: 1-2,000). The types of PA procedures were therapeutic plasma exchange (92% of centers), red cell exchange (86%), leukocyte depletion (87%) and peripheral blood hematopoietic progenitor cell collection (72%). More than 65% of the centers had treated children older than 5 years with PA. Many centers had also performed PA on younger children; 40% have treated patients <12 months of age; 61% had treated patients 1-5 years old. 36% of centers reported that they would perform apheresis regardless of patient weight; 18% used a 5 kg threshold, 11% used 5-10 kg, and 17% used 10 kg as their weight threshold. CONCLUSION: This report is the largest single survey of centers performing PA. The results provide information about the scope and diversity of PA and identify areas where considerable variability in practice exists. Further exploration of these differences could establish best practices in PA through international research and collaboration.


Assuntos
Remoção de Componentes Sanguíneos , Adolescente , Anticoagulantes/uso terapêutico , Doadores de Sangue , Cateteres , Criança , Pré-Escolar , LDL-Colesterol/isolamento & purificação , Circulação Extracorpórea , Humanos , Lactente , Recém-Nascido , Fotoferese
18.
Pediatr Transplant ; 15(3): 334-43, 2011 May.
Artigo em Inglês | MEDLINE | ID: mdl-21492354

RESUMO

We report 23 consecutive pediatric patients with MDS who received allogeneic HSCT on IRB approved protocols between 1992 and 2009 at Children's Memorial Hospital (Chicago, IL). Nine patients had de novo MDS, whereas 14 patients had treatment-related MDS. All patients had a documented cytogenetic abnormality, and monosomy 7/7q- was seen in 12 patients (52%). Fourteen of 23 patients received a myeloablative conditioning regimen; RIC regimens were used for the remaining nine. Five patients relapsed post-transplant, including four patients who received RIC transplant and four patients with treatment-related MDS. For the entire group, estimated five-yr RFS and OS were 47% and 50%, respectively. Treatment-related MDS was associated with decreased RFS in comparison with de novo MDS (33% vs. 70%, p = 0.05). Five-year OS rates reached 80% for those with de novo MDS. RIC regimens were associated with decreased three-yr RFS in comparison with myeloablative regimens (22% vs. 68%, p = 0.02). There was no correlation of survival with blast count at diagnosis, IPSS score, cytogenetic abnormality, donor type, or HLA match. Larger series are needed to confirm prognostic factors so that higher-risk patients can be targeted with novel approaches.


Assuntos
Transplante de Células-Tronco Hematopoéticas/métodos , Síndromes Mielodisplásicas/terapia , Adolescente , Criança , Pré-Escolar , Cromossomos Humanos Par 7 , Citogenética , Intervalo Livre de Doença , Feminino , Humanos , Lactente , Masculino , Pediatria/métodos , Estudos Retrospectivos , Risco , Fatores de Tempo , Condicionamento Pré-Transplante/métodos , Transplante Homólogo/métodos , Resultado do Tratamento
19.
J Clin Apher ; 25(2): 63-9, 2010.
Artigo em Inglês | MEDLINE | ID: mdl-20301140

RESUMO

Apheresis procedures in small children are technically challenging and require special planning with attention to extracorporeal volume. Discontinuous procedures such as extracorporeal photopheresis (ECP) require additional consideration. Alternative methods to perform ECP have been utilized in small children that require manipulation of mononuclear cells outside the standard closed-loop system. We present a safe and feasible alternative to the procedure for children who weigh less than 40 Kg, while maintaining a closed loop, sterile system utilizing the UVAR XTS device. A retrospective chart review was performed analyzing the use of fluid boluses (normal saline in those between 20 and 40 Kg, 5% albumin in those under 20 Kg) before ECP. Eleven patients underwent 334 ECP procedures for acute and chronic graft-versus-host disease (n = 9), and for prevention of graft-versus-host disease (n = 2). Volumes of fluid boluses were calculated based on the expected extracorporeal volume during the first draw cycle. Treatments consisted of at least three draw cycles using the 125 mL bowl. The median weight was 28.5 Kg (range 19 to 39); nine of 11 required red cell transfusions to maintain adequate hematocrit. Complications attributed to ECP included tachycardia, dizziness, nausea, and hypotension; these occurred either in combination or isolation in 31% of the procedures and resolved following additional fluid boluses. Only three (0.8%) required early photoactivation due to these complications. The median time to completion of treatment was 2 h and 58 min (range 1:30 to 5:03). ECP is well tolerated in low-weight pediatric patients if hematocrit and hydration are carefully maintained.


Assuntos
Doença Enxerto-Hospedeiro/terapia , Fotoferese/métodos , Adolescente , Peso Corporal , Separação Celular , Criança , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Fotoferese/efeitos adversos , Fotoferese/instrumentação , Estudos Retrospectivos
20.
Curr Pharm Des ; 14(20): 1987-96, 2008.
Artigo em Inglês | MEDLINE | ID: mdl-18691109

RESUMO

Allogeneic hematopoietic stem cell transplantation (HSCT) may be performed to treat a variety of malignant and non-malignant disorders by eradicating tumor, replacing a non-functioning with a normal immune system, or replenishing a deficient enzyme. While HSCT may provide cure for many patients, barriers such as acute and chronic graft-versus-host disease (a/cGVHD) and graft failure continue to challenge clinicians with considerable potential for morbidity and mortality. A thorough understanding of each disease process is essential to the development of both pharmacologic and non-pharmacologic therapies in this setting; unfortunately, acute and chronic GVHD, are distinct, complex entities, and medications used to prevent and treat them cause significant toxicities and leave patients at high risk for overwhelming infections. Standard pharmacologic therapies that are currently in use are limited in that they have the potential to cause significant toxicity without completely curing the disease. Novel, non-pharmacologic therapies for the prevention and treatment of acute and chronic GVHD must continue to be developed and studied in randomized trials. Given that the potential mechanisms of action of the non-pharmacologic therapies discussed herein attempt to modulate the cellular milieu that supports the development of GVHD, a brief discussion of GVHD and its pathophysiology is warranted; detailed discussions are provided by Cutler et al. and Bolanos-Meade elsewhere in the current issue. We will therefore focus on two non-pharmacological innovative forms of therapy, and potentially, prevention of GVHD.


Assuntos
Doença Enxerto-Hospedeiro/prevenção & controle , Transplante de Células-Tronco Hematopoéticas/métodos , Doença Aguda , Animais , Doença Crônica , Ensaios Clínicos como Assunto , Rejeição de Enxerto/prevenção & controle , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/fisiopatologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Transplante de Células-Tronco Mesenquimais/métodos , Fotoferese/métodos
SELEÇÃO DE REFERÊNCIAS
DETALHE DA PESQUISA