Patient-acceptable symptom state as an outcome measure in the daily care of patients with ankylosing spondylitis.

OBJECTIVE: We assessed the prevalence of patients with ankylosing spondylitis (AS), rating their state as acceptable (patient-acceptable symptom state; PASS), among 190 patients with AS seen in daily practice. Factors associated with PASS status and PASS thresholds for outcome measures were also analyzed. METHODS: The characteristics of patients with affirmative and negative assignment to PASS were compared. Associated factors were estimated by logistic regression models and PASS thresholds by the 75th percentile and receiver-operating characteristic curve methods. RESULTS: A total of 77% of patients rated their state as acceptable (95% CI 62-91). These patients were taking fewer nonsteroidal antiinflammatory drugs and corticosteroids, practiced more exercise, had less anxiety and depression, and had lower values of all patient-reported outcome measures, physicians' assessment, AS Disease Activity Score (ASDAS) and C-reactive protein. Lower values of Bath AS Disease Activity Index and physician's global assessment were independent factors associated with acceptable symptom state. High rates of anxiety and depression were found in patients not in PASS. The thresholds with the 75th percentile approach were 4.55 for the BASDAI and 2.84 for the ASDAS. Fifty-three percent of patients in PASS had a high or very high disease activity state according to ASDAS cutoff values. CONCLUSION: A high percentage of patients with AS in daily practice declared that their symptom state was acceptable. PASS status correlated with physician global assessment and BASDAI. PASS thresholds for common recommended outcome measures were relatively high and many patients in PASS had unacceptably high disease activity states according to ASDAS. Other factors such as psychological problems may influence a negative PASS state.

Pattern of use and safety of non-steroidal anti-inflammatory drugs in rheumatoid arthritis patients. A prospective analysis from clinical practice.

OBJECTIVE: The aim of this study was to determine the prescription pattern and the safety profile of non-steroidal anti-inflammatory drugs (NSAID), including cyclooxygenase-2 inhibitors (COXIB) in patients with rheumatoid arthritis (RA) under a real life clinical setting. PATIENTS AND METHODS: Data was obtained from the EMECAR study, a prospective cohort of randomly selected RA patients (n=789) followed up from 1999 to 2004 in Spain. RESULTS: Upon entry into the cohort, 613 (78%) patients included took or had taken NSAID because of RA. Patients treated with NSAID, compared to those who did not take these compounds, were younger (60 versus 66 years of age) and have had both less cardiovascular (11 versus 20%; p<0.001) and gastric ulcer (11 versus 23%; p<0.001) complications. In the group of patients that used NSAID, RA had been diagnosed earlier (age at onset 47 versus 53; p<0.001) and was more active (DAS28: 4.4 versus 3.7; p<0.001). During follow-up, the percentage of RA patients using NSAID decreased from 78% in year 2000 to 66% in 2004. The use of antiulcer agents increased from 11% in 2000 to 60% in 2004, independently of both the use of classic NSAID or COXIB and the presence of risk factors for NSAID-induced gastropathy. Severe gastric complications and cardiovascular events were infrequent and the incidence was not different between patient who took NSAID and those who did not, as well as between patients treated with classic NSAID or with COXIB. CONCLUSIONS: NSAID are commonly used in the management of RA. These compounds are well tolerated and the frequency of severe adverse events attributed to them is relatively low under daily practice conditions in these patients.

Treatment for children with severe aplastic anemia and sickle cell disease in low income countries in Latin America: a report on the recent meetings of the Monza International School of Pediatric Hematology/Oncology (MISPHO): Part III.

Treatment of hematologic disorders in low-income countries (LIC) is difficult. This report summarizes treatment of sickle cell disease and aplastic anemia by pediatric hematologists from 15 LIC who participate in the Monza International School of Pediatric Hematology/Oncology (MISPHO). Patients with severe sickle cell disease were treated with low dose hydroxyurea, which safely reduced vaso-occlusive crises. Patients with severe aplastic anemia fared poorly due to lack of availability and high cost of anti-thymocyte globulin and cyclosporine and lack of access to stem cell transplantation. Appropriate therapy was most likely to occur in MISPHO centers with an active twinning program with a center in a high-income country.

Uso de la prueba de rupturas cromosómicas en el estudio de la anemia de Fanconi / Use of the chromosome rupture test in the study of Fanconi's anemia. Preliminary results

La anemia de Fanconi es un desorden genético recesivo con ambos patrones de herencia, autosómico y ligado al sexo, caracterizada por diferentes malformaciones congénitas, fallo de médula ósea y una elevada predisposición a desarrollar tumores sólidos y leucemia mieloide aguda. Es una enfermedad monogénica con expresión citogenética dada por inestabilidad cromosómica tanto espontánea como provocada por agentes inductores de enlaces cruzados en las cadenas de ADN. Se presentan 2 pacientes masculinos, hermanos, de 5 y 7 años de edad, con malformaciones congénitas e insuficiencia medular. Se les realizó el estudio de rupturas cromosómicas con el uso del diepoxibutano y se observaron múltiples rupturas y figuras radiales, lo que confirmó el diagnóstico.

Fanconi's anemia is a recurrent genetic disorder with both patterns of heredity, autosomal and linked to sex. It is characterized by different congenital malformations, bone marrow failure and an elevated predisposition to develop solid tumors and acute myeloid leukemia. It is a monogenic disease with cytogenetic expression given by chromosomal instability, both spontaneous and provoked by agents inducing cross-links in the DNA chains. Two male siblings aged 5 and 7 years old, with congenital malformations and medullar insufficiency were presented. The study of chromosome rupture was conducted by using diepoxybutane. Multiple ruptures and radial figures were observed, which confirmed the diagnosis.

Leucemia linfoblástica aguda en niños: resultados del tratamiento en 24 años / Acute lymphoblastic leukemia in children: result of treatment in twenty four years

Se realiza un estudio de 186 niños diagnósticados de leucemia linfoblástica aguda (LLA) de 1972 a 1995 y tratados con distintos esquemas terapéuticos, casi todos incluidos en los protocolos del grupo Latinoamericano de Tratamiento de Hemopatías Malignas (GLATHEM). Los pacientes se clasificaron en 5 grupos. El grupo está constituido por 20 niños tratados antes de iniciarse en nuestro centro los estudios del GLATHEM; el grupo 2 corresponde a 94 pacientes tratados con los protocolos clásicos de la década de los 70 grupos 3,4 y 5 fueron tratados con esquemas agresivos tipo BFM a partir de 1982. En los grupos 4 y 5 se incluyó el Methotrexate Endovenoso en altas dosis. Independientemente de que se había utilizado distintos parámetros para la clasificación por grupos pronósticos en los protócolos del GLATHEM, con vistas a lograr uniformidad en este trabajo se clasificaron a todos los pacientes retrospectivamente en 3 grupos: bueno, intermedio y malo, en base a la edad y la cifra de leucocitos. Se obtuvo remisión completa en el 92 por ciento de los pacientes incluidos en el GLATHEM sin diferencias significativas entre los distintos protócolos. La sobrevida global se elevó desde un 15 por ciento a los 5 años en el grupo 1 hasta un 70 por ciento en los grupos 4 y 5. La sobrevida libre de eventos (SLE) se eleva de 31 por ciento en el grupo 2 a 67 por ciento en los grupos 4 y 5 en conjunto. El grupo 5 considerado aisladamente tiene un 88 por ciento de sobrevida global y un 72 por ciento de SLE a los 3 años de evolución. Se encontró una diferencia significativa en los porcentajes sobrevida entre los grupos bueno, intermedio y malo en los protocolos de la década de los 70, mientras en los esquemas tipo BFM se borran las diferencias entre bueno e intermedio, persistiendo una sobrevida significativamente menor en el grupo de pronóstico malo. Los porcentajes de sobrevida de 5 años para estos esquemas fueron de 81 por ciento, 78 por ciento y 26 por ciento para los grupos de riesgo bueno, intermedio y malo respectivamente. Los factores pronósticos analizados en forma aislada que tuvieron importancia significativa en los porcentajes de sobrevida fueron: la edad, la esplenomegalia y la cifra de leucocitos superior a 50x10 a la 9/l

Cessation of therapy in chilhood acute lymphoblastic leukemia / Suspension de la terapia de leucemia limfoblastica aguda en niños

Two hundred and seventy patients with Acute Lymphoblastic Leukaemia in the pediatric age were studied and treated during 1969-1993 with different therapeutical shemes which virtually all included in the protocols of the Latin America Group for the Treatment of Malignant Hemopathics (GLATEM). These patients have been treated in different hospitals of Cuba, and most of them were in first complete remission. Of the total of patients, 8 have been treated before GLATEM protocols; 92 were treated with schemes of the 70s and the remaining ones oncluded in more recent protocols. treatment, and they were followed during several years with clinical and hematological control. Prognostic group classification was carried out mainly in relation to age and leukocyte count., consideranting thre groups: Good, Intermediate and Poor. Percentage of relapse, site, and time of occurrence are discussed, and they are related to pronostic factors at the beginning of the dosease. Fourty-two (15.5 por ciento) patients have relapsed, mainly in bone marrow during an observation period of 0-21 years off therapy (mean 8 years). Half of relapses occurred during the first year following of therapy and only two after 4 years off therapy, which represents 1por ciento of all patients. More late relapses significantly occurred in male patients and in patients with notable hepatomegaly at the beginning of the disease. Survival probability form the whole group is 90 per cent al 5 years and 84 per cent at 10 years after cessation of therapy. For patients relapsing off therapy, probability of survival in 5 years is 53 per cent. The CNS relapses being of the best course and those of bone marrow of the most unfavorable one. An import number of patients had their therapy stopped a second time. The hihg number of patients, event-free for several years without treatment; show that true cures can be attained in ALL children