El trasplante de hígado es un procedimiento plausible, efectivo y seguro en pacientes con hemofilia: caso clínico / Liver transplantation in hemophilia A: report of one case

Due to blood derivative requirements, many patients with hemophilia were exposed to Hepatitis C virus infection (HCV) before the availability of HCV testing. We report a 46-year-old male with Hemophilia A with a hepatitis virus C infection since 2004 causing a cirrhosis. Due to a hepatopulmonary syndrome, he received a liver allograph using a factor VIII replacement protocol, after eradicating the virus C. He had a good postoperative evolution, and no more factor VIII was required after transplantation until his last assessment.

Hemorragia intracraniana espontânea em pediatria: relato de paciente hemofílico que sobreviveu devido a cisto cerebral / Spontaneous intracranial hemorrhage in children: report of a hemophilia patient who survived due to a brain cyst

RESUMO Relatamos o caso de um uma criança de 2 anos de idade que sobreviveu após um episódio agudo de hemorragia intracraniana espontânea grave com sinais clínicos e radiológicos de hipertensão intracraniana e herniação transtentorial. O paciente foi para cirurgia de urgência para drenagem do hematoma, sendo inserido um cateter para monitorar a pressão intracraniana. Na análise da tomografia de crânio inicial, antes da drenagem do hematoma, constatou-se um cisto cerebral contralateral ao hematoma que, segundo análise do neurocirurgião e do neuroradiologista, possivelmente evitou um desfecho pior, visto que o cisto serviu de acomodação para o cérebro após a hemorragia maciça. Após investigação, constatou-se tratar de um caso de hemofilia tipo A sem diagnóstico prévio. O paciente foi tratado em terapia intensiva com controle da pressão intracraniana, reposição de fator VIII e obteve alta sem sequelas neurológicas evidentes.

ABSTRACT We report the case of a 2-year-old child who survived an acute episode of severe spontaneous intracranial hemorrhage with clinical and radiological signs of intracranial hypertension and transtentorial herniation. The patient underwent emergency surgery to drain the hematoma, and a catheter was inserted to monitor intracranial pressure. In the initial computed tomography analysis performed prior to hematoma drainage, a brain cyst was evident contralateral to the hematoma, which, based on the analysis by the care team, possibly helped to avoid a worse outcome because the cyst accommodated the brain after the massive hemorrhage. After the investigation, the patient was determined to have previously undiagnosed hemophilia A. The patient underwent treatment in intensive care, which included the control of intracranial pressure, factor VIII replacement and discharge without signs of neurological impairment.

Complicações do parto e resultados perinatais em gestantes portadoras da doença de von Willebrand / Delivery complications and perinatal results in pregnant women with von Willebrand disease

OBJETIVO: Analisar as complicações maternas associadas ao parto e puerpério, bem como os resultados perinatais, em gestantes portadoras da doença de von Willebrand. MÉTODOS: Foram analisados os prontuários de todas as gestantes com diagnóstico de doença de von Willebrand, cujo parto foi realizado nesta instituição no período compreendido entre março de 2001 e agosto de 2007. Foram investigadas características relativas à via de parto, incidência de complicações hemorrágicas no parto e no puerpério imediato, perfil dos exames laboratoriais maternos e resultados perinatais. As variáveis foram estudadas descritivamente, calculando-se freqüências absolutas e relativas, médias, medianas e desvios padrão. RESULTADOS: Foram revisadas 13 gestações em oito pacientes portadoras da doença. No período ocorreram 13.037 partos na instituição, perfazendo incidência de 0,1 por cento. Seis pacientes (75 por cento) apresentavam o tipo 1 da doença, e duas (25 por cento) o tipo 2. No terceiro trimestre, a média da atividade do fator VIIIc foi de 98,5 por cento. A cesárea foi realizada em nove casos (69 por cento), cuja anestesia foi do tipo raquidiana em sete casos. Como complicação do parto, um caso evoluiu com descolamento prematuro da placenta e foi realizada a cesárea. Um caso apresentou sangramento no primeiro pós-parto, necessitando reposição do fator VIII. Em dois casos foi realizada a reposição profilática do fator VIII antes da cesárea. A restrição do crescimento fetal ocorreu em cinco casos (38,5 por cento). O peso dos RNs apresentou média de 2676 gramas e um caso (7,8 por cento) apresentou Apgar de 1º minuto inferior a sete. CONCLUSÃO: O parto em gestantes com diagnóstico de doença de von Willebrand tem evolução favorável quando cuidados são tomados procurando oferecer assistência específica. O crescimento fetal deve ser monitorizado nessas gestantes.

OBJECTIVE: To study maternal complication associated to delivery and the puerperium period in pregnancies affected by von Willebrand's disease. METHODS: Chart data of all the pregnant women with diagnosis of von Willebrand disease were retrospectively reviewed. All cases with von Willebrand's disease that had given birth at this institution, between March 2001 and August 2007, were analyzed. The following variables were investigated: mode of delivery, hemorrhage complications during delivery and postpartum, maternal blood exams and perinatal results. Variables were studied descriptively, using absolute and relative frequencies, means, medians and standard deviations. RESULTS: 13 pregnancies of eight women with the disease were reviewed. During this sane period, there were 13,037 deliveries in the institution, resulting in an incidence of 0.1 percent. Six women (75 percent) were type 1 disease and, two (25 percent) were type 2. The last Factor VIIIc activity presented a mean value of 98.5 percent. A Cesarean section was performed in nine pregnancies, with epidural anesthesia in seven. Delivery complication occurred in two cases: one presented placental abruption and a Cesarean was performed. The other, presented postpartum hemorrhage in the first day and required reposition with factor VIII. Two cases received factor VIII before Cesarean section. Fetal growth restriction was detected in five pregnancies (38.5 percent). Mean birth weight was of 2676 grams and one case presented 1st minute Apgar score below seven. CONCLUSION: Delivery in patients with von Willebrand disease has a favorable evolution when specific assistance is provided. In these pregnancies,fetal growth should be monitored.

Diagnosis and treatment of congenital hemophilia with inhibitors: A Latin American perspective / Diagnóstico y tratamiento de la hemofilia congénita con inhibidores: Una perspectiva latinomericana

The Committee of Latin America on the Therapeutics of Inhibitor Groups (CLOTTING) is composed of a number of hemophilia specialists from Latin America. The group aims to encourage the adoption of a good standard of care for Latin American patients with hemophilia. The occurrence of inhibitors in patients with hemophilia poses clinical challenges, and it is estimated that between 1000 and 3 000 patients in Latin America are affected by hemophilia with inhibitors. There is an urgent need to establish a regional consensus and clinical guidelines for the diagnosis and treatment of these patients. We present an extensive review based on best current clinical practice and published literature, as seen from a Latin American perspective, taking into account the variable nature of hemophilia care available in the various countries in this Region.

El Comité Latinoamericano sobre la Terapéutica de Personas con Inhibidores (CLOTTING) está compuesto por un grupo de especialistas en hemofilia de Latinoamérica. El objetivo del grupo es promover la adopción de un estándar de tratamiento óptimo para los pacientes con hemofilia en Latinoamérica. La prevalencia de inhibidores en pacientes con hemofilia en Latinoamérica determina desafíos clínicos y se estima que de 1000 a 3000 pacientes en esta región están afectados con hemofilia e inhibidores. Existe una necesidad urgente de establecer un consenso regional y guías clínicas para el diagnóstico y tratamiento de estos pacientes. Nosotros presentamos una revisión exhaustiva basada en las mejores prácticas clínicas vigentes y en los datos publicados en la literatura, con una perspectiva latinoamericana, tomando en cuenta la variabilidad existente de los tratamientos de la hemofilia disponibles en los diferentes países de esta Región.

Lyophilized cryoprecipitate for children with hemophilia A.

Cryoprecipitate is the most cost-effective treatment for bleeding events in hemophilia A children in Thailand. Lyophilized cryoprecipitate (LC), a freeze-dried heat-treated blood product from the National Blood Center, can be stored in a refrigerator. The objectives of this study were to assess the efficacy of LC in bleeding episodes of children with hemophilia A and to determine the pharmacokinetics of single dose LC infusion. Eight male children with congenital factor VIII deficiency, aged 1.2-13 years, with 12 active bleeding episodes were infused with reconsitituted LC (average factor VIII activity = 236 IU per bottle) at the dose of 1 bottle per 11 kg. All patients achieved clinical hemostasis by 12 hours post-infusion. The Factor VIII activity reached peak level within the first hour and declined slowly in the subsequent hours and retained in vivo Factor VIII activity at 27.3 +/- 5.6 per cent at 12 hours post-infusion. The incremental factor VIII activity was 2.37 +/- 1.05 per cent (mean +/- SD) per U infused per kilogram body weight. The half-life of infused factor VIII was 10.5 hours. The authors concluded that reconstituted LC infusion is efficacious in the treatment of bleeding episodes in children with hemophilia. Cost-effectiveness of this product and cryoprecipitate and other purified factor VIII should be evaluated.

Haemostasis with cryoprecipitate in patients undergoing surgery for severe von Willebrand disease.

BACKGROUND: There are limited data on the management of haemostasis in patients with severe von Willebrand disease undergoing major surgery. Data on the use of cryoprecipitate in this setting are even more limited. In many developing countries cryoprecipitate is often the only available source of factor replacement. The minimum factor levels required for maintaining haemostasis after surgery have never been carefully evaluated. METHODS: Data from 3 patients with severe von Willebrand disease who underwent 4 major surgical procedures at our institution, using lower than standard recommended doses of cryoprecipitate were analysed for adequacy of factor replacement and complications. RESULTS: The average preoperative cryoprecipitate infusion was 22.5 i.u. of factor VIII/kg (range: 15-25). The bleeding time done by the modified Ivy method, 30 minutes after infusion, was normal in all these patients. The average cryoprecipitate support for days 1-3 was 16.5 i.u. of factor VIII/kg/day (range: 12.5-25) and for days 4-10 was 12.4 i.u. of factor VIII/kg/day (range: 8.3-16). The mean duration of factor replacement was 12 days (range: 7-17). Two patients had delayed bleeding, one on day 3 attributed to the inadvertent use of a non-steroidal anti-inflammatory drug and the second on day 10 which was probably secondary to septicaemia. Bleeding resolved in both these patients as soon as the precipitating factors were relieved. CONCLUSION: The total amount of factor replaced in our patients is approximately half of what would have been used if the usual recommendations were followed. The data suggests that lower doses of cryoprecipitate could be adequate for major surgery and wound healing in severe von Willebrand disease. This will lead to lowering of costs and reducing the risk of transfusion-associated virus infection.

Hematoma intracerebral como primera manifestación de hemofilia / Intracranial hematoma as the first manifestation of hemophilia

La hemorragia intracraneana ocurre entre 2 por ciento y 13 por ciento de los enfermos hemofílicos,pudiendo ser subaracnoidea, subdural o intracerebral (hematoma intracerebral). Es infrecuente como primera manifestación de la enfermedad, habiéndose demostrado en 5,9 por ciento de los pacientes con hemofilia A. En 54 por ciento de los casos hay antecedentes de traumatismo, pero en 38 por ciento no se identifica una causa desencadenante. La tomografía axial computadorizada suele permitir el diagnóstico sin dificultad, pero excepcionalmente las imágenes obtenidas pueden ser confundidadas con las de otras afecciones. Se describe un niño de tres meses de edad, en quien la primera manifestación de hemofilia fue un hematoma intracerebral, interpretado inicialmente como tumor, descubriéndose su verdadera naturaleza durante la intervención quirúrgica y su etiología hemofílica al estudiar la coagulación como consecuencia de nuevos sangramientos intracraneanos en el período postoperatorio

Prophylactic treatment for hemophilia A patients: a pilot study.

Prophylactic treatment with factor VIII concentrate was given to six hemophilia A boys whose factor VIII:C ranged from 1% to 3.5% at Ramathibodi Hospital. The age ranged from 11 to 16 years with the median age of 12 years old. Each patient received factor VIII concentrate twice a week in the dosage of 8-10 unit per kg for one year. During the prophylactic period, bleeding episodes seldom occurred. They did not need hospitalization. The absence from school was reduced. They became muscular from regular daily exercise. They could join the activity at school and lived a near normal life. The patients and family were very happy since they did not have to worry about bleeding. No adverse effect was found. The only constraint was the cost. It cost 180,000 baht (US$ 7,200) per year or 15,000 baht (US$ 600) per month for a 25 kg hemophiliac boy.