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INTRODUCTION: Prompt, appropriate coagulation factor replacement according to injury and bleeding severity in persons with haemophilia is required to prevent acute and long-term complications. AIMS: Increase proportion of persons with haemophilia A (HA) and B (HB) treated appropriately for an acute injury and bleeding episode at a tertiary children's emergency department (ED) from 65% to 85% and sustain for one year. Secondary aim: increase time interval between patient ED encounters with out-of-range factor dosing. METHODS: Utilizing quality improvement methodology and plan-do-study-analyze cycles, ED encounters for individuals with HA/HB receiving coagulation factor replacement for injuries were audited for in-range coagulation factor dosing. Goal factor dose defined as 50% correction for minor bleeds and 100% correction for major bleeds. Optimal dosing range defined as 90%-120% of the calculated goal dose to account for vial size variability. Interventions targeted communication via the EMR problem list and optimization of physician education. RESULTS: Our previous publication demonstrated 33.3% of ED encounters with out-of-range factor replacement. Following several interventions, the cumulative rate of encounters with out-of-range dosing decreased to 18%. Overall, there was an increase in the mean percent of encounters receiving optimal factor dosing for both HA/HB compared to baseline (82.2% vs. 71.1%), though this was not a statistically significant difference. CONCLUSION: Despite implementation of multiple interventions, out-of-range factor dosing continues to occur. Our team plans to reinstate simulation center education for ED staff and continue education efforts of pharmacists and hematology trainees with the goal of further reducing out-of-range dosing in our ED.
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Hemofilia A , Melhoria de Qualidade , Criança , Humanos , Fatores de Coagulação Sanguínea/uso terapêutico , Hemofilia A/tratamento farmacológico , Hemorragia/etiologia , Serviço Hospitalar de EmergênciaRESUMO
BACKGROUND: Ε-Aminocaproic acid oral solution (EACA OS) is the only commercially available antifibrinolytic for patients who cannot swallow tablets. Insurance denials and high costs remain barriers to its use. OBJECTIVES: To determine the safety and efficacy of crushed tranexamic acid tablets in water (cTXAw) for children with bleeding disorders. METHODS: We retrospectively reviewed records of children (<10 years) with bleeding disorders who received cTXAw or EACA OS from 1 December 2018, through 31 July 2022, at Mayo Clinic (Rochester, Minnesota). Bleeding outcomes were defined according to ISTH criteria. RESULTS: Thirty-two patients were included (median age, 3 years; male, n = 23). Diagnoses were VWD (n = 17), haemophilia (n = 5), FVII deficiency (n = 3), inherited platelet disorder (n = 4), ITP (n = 2), and combined FV and FVII deficiencies (n = 1). Thirty-two courses of cTXAw (monotherapy 24/32; mean duration 6 days) and fifteen courses of EACA (monotherapy 12/15; mean duration 5 days) were administered. No surgical procedures (n = 28) were complicated by bleeding. Of the 19 bleeding events, 16 had effective haemostasis, two had no reported outcome, and one had no response. cTXAw and EACA were equally effective in preventing and treating bleeding (p value > .1). No patients had adverse effects. Eight of 19 patients (42%) who were initially prescribed EACA OS did not receive it because of cost or insurance denial. The estimated average wholesale price of one treatment was $94 for cTXAw and $905 for EACA OS. CONCLUSIONS: CTXAw appears to be an effective, safe, and low-cost alternative option to EACA OS for young children with bleeding disorders.
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Ácido Tranexâmico , Humanos , Ácido Tranexâmico/uso terapêutico , Ácido Tranexâmico/administração & dosagem , Masculino , Pré-Escolar , Feminino , Criança , Estudos Retrospectivos , Comprimidos , Lactente , Antifibrinolíticos/uso terapêutico , Antifibrinolíticos/administração & dosagem , Água , Hemorragia/tratamento farmacológico , Transtornos da Coagulação Sanguínea/tratamento farmacológicoRESUMO
The presentation of immune thrombocytopenia is dependent on the degree of thrombocytopenia, with no to mild bleeding symptoms, primarily mucocutaneous bleeding. Severe bleeding in other organ systems is a rare complication. Spontaneous hemarthrosis is rare in patients without hemophilia. We report a child presenting with oral and cutaneous petechial lesions and left knee hemarthrosis without trauma. Laboratory findings showed severe thrombocytopenia consistent with immune thrombocytopenia. Serologic tests were consistent with Lyme disease. Hemarthrosis was presumed secondary to Lyme disease monoarticular joint inflammation with bleeding exacerbated by severe thrombocytopenia. Hemarthrosis resolved and platelet counts normalized following immunoglobulin infusion, steroid course, and antibiotics.
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Hemofilia A , Doença de Lyme , Púrpura Trombocitopênica Idiopática , Humanos , Criança , Hemartrose/complicações , Hemartrose/diagnóstico , Púrpura Trombocitopênica Idiopática/complicações , Doença de Lyme/complicações , Doença de Lyme/diagnóstico , Doença de Lyme/tratamento farmacológico , Hemofilia A/complicações , Antibacterianos/uso terapêuticoRESUMO
BACKGROUND: This study was performed to describe the single-center experience of deep vein thrombosis (DVT) in children with severe traumatic brain injury (sTBI) who were mechanically ventilated with a central line, and to identify potentially modifiable risk factors. It was hypothesized that children with DVT would have a longer duration of central venous line (CVL) and a higher use of hypertonic saline (HTS) compared to those without DVT. PROCEDURE/METHODS: This was a retrospective study of children (0-18 years) with sTBI, who were intubated, had a CVL, and a minimum intensive care unit (ICU) stay of 3 days. Children were analyzed by the presence or absence of DVT. HTS use was evaluated using milliliter per kilogram (ml/kg) of 3% equivalents. Univariable and multivariable logistic regression models were used to determine which factors were associated with DVT. RESULTS: Seventy-seven children met inclusion criteria, 23 (29.9%) had a DVT detected in an extremity. On univariable analysis, children with DVT identified in an extremity had prolonged CVL use (14 vs. 8.5 days, p = .021) and longer duration of mechanical ventilation (15 vs. 10 days, p = .013). HTS 3% equivalent ml/kg was not different between groups. On multivariable analysis, mechanical ventilation duration was associated with DVT detection in an extremity, whereas neither CVL duration nor HTS use had an association. CONCLUSIONS: There was a high incidence of extremity DVT detected in children with sTBI who received invasive mechanical ventilation and had a CVL. HTS administration was not associated with DVT detection in an extremity.
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Lesões Encefálicas Traumáticas , Cateteres Venosos Centrais , Trombose Venosa , Criança , Humanos , Estudos Retrospectivos , Trombose Venosa/etiologia , Trombose Venosa/epidemiologia , Cateteres Venosos Centrais/efeitos adversos , Incidência , Fatores de Risco , Lesões Encefálicas Traumáticas/complicaçõesRESUMO
BACKGROUND: Evidence-based anticoagulation programs usually serve a local, adult patient population. Here we report outcomes for a regional combined pediatric-adult program. AIMS: The aims of this study were: (1) Compare the pre- vs. post-implementation quality of therapy (% time in therapeutic range (%TTR) and compliance). (2) Assess anticoagulant-relevant outcomes (bleeding and thrombotic complications). METHODS: Data were collected for the years 2014-2019. Rosendaal linear interpolation was used to calculate %TTR. Bleeding complications were categorized using ISTH-SSC standard nomenclature and new thrombotic events were reviewed. RESULTS: The patients were divided into a long-term warfarin group (N = 308), 80.2% of whom had cardiac-related therapeutic indications (median age 24y), and a second group (N = 114) comprised of short-term and non-warfarin long-term anticoagulation (median age 16y). Median %TTR for those on long-term warfarin was 78.9%. The incidence of major and clinically relevant non-major bleeding events was 1.65 and 2.43 /100 person-years of warfarin use, respectively. Thromboembolism (TE) incidence was 0.78/100 patient-years of warfarin use. Neither bleeding nor thrombosis was associated with %TTR (p = 0.48). Anticoagulant indication was the only variable associated with bleeding risk (p = 0.005). The second group had no on-therapy TE events but 7.9% experienced bleeding. Complete data were available for a randomly sampled pre-program warfarin group (N = 26). Median %TTR improved from 17.5 to 87% pre- vs. post-implementation. Similarly, compliance (defined as ≥ 1 INR/month) improved by 34.3%. CONCLUSIONS: In conclusion, this program significantly improved and sustained %TTR and compliance. The lack of association between bleeding and thrombosis events and %TTR may be related to the high median %TTR (> 70%) achieved by this approach.
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Tromboembolia , Trombose , Humanos , Criança , Adulto , Adulto Jovem , Adolescente , Coeficiente Internacional Normatizado , Varfarina/efeitos adversos , Anticoagulantes/efeitos adversos , Coagulação Sanguínea , Hemorragia/induzido quimicamente , Hemorragia/tratamento farmacológico , Tromboembolia/tratamento farmacológico , Trombose/tratamento farmacológico , Resultado do TratamentoRESUMO
Patients with sickle cell disease (SCD) have a high risk for venous thromboembolism which is associated with increased risk of mortality. Studies examining risk of pulmonary embolism (PE) in children with SCD are lacking. This study was conducted in children with SCD between 0-21 years of age using a nationwide administrative database in the United States- Pediatric Health Information System (PHIS) from January 2010 to June 2021. Diagnostic codes and imaging, procedure, and pharmaceutical billing codes were used to identify PE and potential clinical, demographic, and utilization risk factors. Logistic regression analyses were performed to assess association between risk factors and PE. We identified 22,631 unique patients with SCD with a median age of 10.8 years (range: <0.1-20.9). A total of 120 (0.53%) patients developed a PE with median age of 17.4 years (range: 6.6-20.9) at PE diagnosis. Patients with PE had longer hospitalization and more frequent ICU admissions than patients without PE (p < 0.001). Risk factors significantly associated with PE on multivariable analysis included older age, prior history of central venous line (CVL), acute chest syndrome, and apheresis. Mortality was not significantly different between those with and without PE. The prevalence of PE in hospitalized children with SCD was estimated to be 0.53%. Patients with PE had higher healthcare utilization characteristics. Factors significantly associated with PE suggest that the risk for PE in SCD may be related to the severity of disease state. Future trials are needed for risk stratification and PE prevention strategies in children with SCD.
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Anemia Falciforme , Embolia Pulmonar , Criança , Humanos , Estados Unidos/epidemiologia , Lactente , Pré-Escolar , Adolescente , Adulto Jovem , Adulto , Prevalência , Anemia Falciforme/complicações , Anemia Falciforme/epidemiologia , Embolia Pulmonar/epidemiologia , Embolia Pulmonar/etiologia , Fatores de Risco , Hospitalização , Estudos RetrospectivosRESUMO
BACKGROUND: Coagulopathy and thrombosis are well-described complications of asparaginase therapy. However, treatment practices in pediatric hematology/oncology (PHO) patients vary widely as evidence-based guidelines for clinical management of these complications in this population are lacking. OBJECTIVE: The objective of this study was to assess management practices of asparaginase-related coagulopathy by pediatric hematologist/oncologist attending physicians. DESIGN/METHOD: Email survey sent to 2327 PHO physicians primarily practicing in the United States. RESULTS: Two hundred eighty-five (12.2%) attending physicians completed the survey. Only 4.6% (n=13/285) routinely prescribe prophylactic anticoagulation during induction chemotherapy for leukemia. Slightly more than half (n=145/250, 50.9%) of all providers perform baseline coagulation studies. Most providers that were surveyed (n=185/285, 64.9%) only replete coagulant factors if the patient experiences bleeding or bruising. One hundred thirty (n=130/285, 45.6%) physicians replace low fibrinogen. The median fibrinogen replacement was 100 mg/dL (range: 40 to 200 mg/dL) with the median target of at least 100 mg/dL (range: 50 to 200 mg/dL). A minority of physicians (n=39/250, 13.7%) replace low antithrombin. The median antithrombin cutoff activity level was 60% (range: 40% to 100%) with a median target of 75% (range: 40% to 125%). CONCLUSIONS: There is a significant variation in PHO physician practices for monitoring and management of asparaginase-associated hemostatic derangements. Evidence-based guidelines have the potential to standardize practices.
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Transtornos da Coagulação Sanguínea , Oncologistas , Leucemia-Linfoma Linfoblástico de Células Precursoras , Criança , Humanos , Asparaginase/uso terapêutico , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Transtornos da Coagulação Sanguínea/tratamento farmacológico , Fibrinogênio/uso terapêutico , Antitrombinas/uso terapêutico , Anticoagulantes/uso terapêutico , Antitrombina III/uso terapêuticoRESUMO
An innovative approach to anticoagulation management during the COVID-19 pandemic was used at our center that allowed patients to stay in their vehicle while our anticoagulation advanced practice registered nurse obtained blood for point-of-care international normalized ratio (INR) testing while education and counseling were completed. A significant improvement in the median percentage of INR within the therapeutic range was observed among the patients who used the drive-through clinic. A small group of patients improved compliance to anticoagulation monitoring. Clinical care models, such as this clinic approach may improve patient compliance and adherence to anticoagulation beyond the pandemic needs.
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BACKGROUND: Hemolytic disease of the fetus and newborn (HDFN) attributable to anti-M is rare, although case reports implicate anti-M in varying severities of HDFN, including fetal hydrops and intrauterine death. CASE DESCRIPTION: We describe the case of a newborn with HDFN associated with an atypical immunoglobulin (Ig) G anti-M that reacted best at cold temperatures. The maternal antibody detected in pregnancy was not reactive at 37°C, and a direct antiglobulin test (DAT) on red blood cells (RBCs) from the newborn was negative, suggesting an anti-M that should not have been clinically relevant. However, the infant developed hyperbilirubinemia (bilirubin level, 17.6 mg/dL), hemolytic anemia (hemoglobin nadir, 5.5 g/dL), and reticulocytopenia. Laboratory testing demonstrated the presence of an IgG anti-M in maternal and neonatal samples reacting best at 4°C. This passively acquired IgG anti-M provoked hemolytic anemia in the infant and likely suppressed erythropoiesis, resulting in reticulocytopenia with prolonged anemia. He was treated for IgG anti-M HDFN with 10 intravenous Ig infusions and 10 days of oral prednisone followed by a taper. He required seven transfusions with M- RBCs. His hemoglobin level normalized at 3 months of age. Follow-up at 2 years revealed no hematologic or neuro-developmental concerns. CONCLUSION: To our knowledge, this is the second report of HDFN attributable to an IgG anti-M reacting preferentially at cold temperature with no 37°C reactivity. Clinically relevant IgG anti-M may elude standard testing. Early recognition and testing for cold-reacting IgG anti-M should be considered for newborns with hemolysis, a negative DAT, and prolonged anemia.
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Anemia Hemolítica/imunologia , Eritroblastose Fetal/diagnóstico , Eritroblastose Fetal/imunologia , Imunoglobulina G/sangue , Anemia Hemolítica/complicações , Anemia Hemolítica/tratamento farmacológico , Anemia Hemolítica/etiologia , Transfusão de Sangue , Temperatura Baixa , Teste de Coombs , Eritroblastose Fetal/tratamento farmacológico , Eritroblastose Fetal/etiologia , Eritrócitos/imunologia , Eritropoese/imunologia , Feminino , Hemoglobinas/metabolismo , Humanos , Recém-Nascido , Masculino , GravidezRESUMO
Venous thromboembolism (VTE) is a life-threatening complication rarely encountered with the use of combined oral contraceptives (COCs). Obesity is an additional thrombosis risk factor that has been shown to further increase the risk of VTE with the use of COCs. We present 5 cases of obese adolescents (body mass index >30 kg/m2) who encountered thrombosis complications while on COCs. Although the absolute risk of VTE events in the setting of COCs is rare, caution should be observed when choosing hormonal therapy and safer COCs alternatives discussed with adolescents who are obese.
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Índice de Massa Corporal , Anticoncepcionais Orais/efeitos adversos , Obesidade Infantil/complicações , Tromboembolia Venosa/patologia , Adolescente , Criança , Feminino , Humanos , Prognóstico , Fatores de Risco , Tromboembolia Venosa/induzido quimicamenteRESUMO
INTRODUCTION: Hemostatic prophylaxis (HP) is recommended for patients with bleeding disorders (PWBD) before invasive procedures. However, evidence-based guidelines are needed to determine optimal HP strategies. AIM: To determine outcomes of HP for PWBD undergoing colonoscopy. METHODS: We undertook a retrospective cohort study of HP and outcomes of colonoscopy procedures performed between 9 November 1993 and 13 February 2018 for PWBD who received care in the Mayo Clinic Comprehensive Hemophilia Treatment Center. RESULTS: During the study period, 73 PWBD (58 with milder phenotypes: haemophilia, von Willebrand disease [subtypes 1 and 2; II, VII and XI deficiency]) underwent 141 procedures. Preprocedural HP was given to 61%, and interventions were performed in 47%. Of the 39% without preprocedural HP, postprocedural HP was given for 11%. One major (0.7%; 6 days postprocedure despite HP) and 10 minor (7%) bleeding complications occurred, which tended to be in patients with severe disease and/or after excision of larger polyps. There was no significant difference in the rate of bleeding complications with or without preprocedural HP (8.1% vs 5.5%, respectively; P = .74, Fisher's exact test). CONCLUSION: The low bleeding rates in our cohort suggest that preprocedure HP may be withheld for patients with mild bleeding disorders who undergo colonoscopy with a low likelihood of requiring an intervention or who require only low-risk intervention. This strategy may be best used in experienced centres, provided optimal local hemostasis measures are undertaken and postprocedural HP is rapidly available if high-risk intervention is required. Further studies are needed to determine optimal evidence-based HP strategies for PWBD undergoing colonoscopy.
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Colonoscopia/métodos , Hemorragia/prevenção & controle , Hemostáticos/uso terapêutico , Adulto , Idoso , Estudos de Coortes , Feminino , Hemostáticos/farmacologia , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Resultado do TratamentoRESUMO
Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy. Advancements in technology that enhance our understanding of the biology of the disease, risk-adapted therapy, and enhanced supportive care have contributed to improved survival rates. However, additional clinical management is needed to improve outcomes for patients classified as high risk at presentation (eg, T-ALL, infant ALL) and who experience relapse. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for pediatric ALL provide recommendations on the workup, diagnostic evaluation, and treatment of the disease, including guidance on supportive care, hematopoietic stem cell transplantation, and pharmacogenomics. This portion of the NCCN Guidelines focuses on the frontline and relapsed/refractory management of pediatric ALL.
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Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas/métodos , Oncologia/normas , Recidiva Local de Neoplasia/terapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Fatores Etários , Protocolos de Quimioterapia Combinada Antineoplásica/farmacologia , Criança , Resistencia a Medicamentos Antineoplásicos , Medicina Baseada em Evidências/normas , Humanos , Lactente , Oncologia/métodos , Terapia de Alvo Molecular/normas , Recidiva Local de Neoplasia/diagnóstico , Recidiva Local de Neoplasia/mortalidade , Organizações sem Fins Lucrativos/normas , Leucemia-Linfoma Linfoblástico de Células Precursoras/diagnóstico , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidade , Programa de SEER/estatística & dados numéricos , Taxa de Sobrevida/tendências , Transplante Homólogo , Resultado do Tratamento , Estados Unidos/epidemiologiaRESUMO
The development of factor VIII inhibitors remains a significant clinical challenge in the management of hemophilia A. We present a patient of mixed ethnicity with severe hemophilia A who was found to have a F8 gene hemizygous c.5815G>T mutation resulting in an Ala1939Ser substitution (Ala1920Ser in legacy nomenclature) and possible splice site change that has been reported in only 1 patient previously. He developed an inhibitor shortly after starting replacement recombinant factor VIII (Advate®; Baxalta, Bannockburn, IL, USA) and was successfully treated with immune tolerance therapy. Our report describes the second patient reported to have severe hemophilia due to this mutation and the only case of a factor VIII inhibitor associated with this mutation.
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Inibidores dos Fatores de Coagulação Sanguínea/sangue , Fator VIII , Hemofilia A , Mutação de Sentido Incorreto , Substituição de Aminoácidos , Fator VIII/administração & dosagem , Fator VIII/genética , Hemofilia A/sangue , Hemofilia A/tratamento farmacológico , Hemofilia A/genética , Humanos , Recém-Nascido , MasculinoRESUMO
Pediatric hematologists/oncologists face complex situations such as breaking bad news, treatment/clinical trials discussions, and end-of-life/hospice care. With increasing diversity in patient and physician populations, cultural competency and sensitivity training covering different aspects of pediatric hematology/oncology (PDHO) care can help improve health care delivery and reduce disparities. Though it is considered a required component of fellowship training, there is no clearly defined curriculum meant specifically for PDHO fellows-in-training (PDHO-F). A national online survey of 356 PDHO-F and 67 PDHO program directors (PDHO-PD) was conducted to assess the educational experience, perceptions about identifying barriers including one's own biases and trainee comfort in delivering culturally sensitive care in various PDHO relevant clinical situations. One hundred and eleven (31.2%) PDHO-F and 27 (40.3%) PDHO-PD responded. 30.6% of PDHO-F "strongly agreed/agreed" they received comprehensive cross-cultural communication (CCC) training. The top two teaching methods were faculty role modeling and informal teaching. Majority of CCC training is in medical school or residency and only 10.8% of PDHO-F reported that most of their CCC training was in fellowship. In most clinical situations, there was a modest direct correlation between the fellow's level of agreement that they received comprehensive CCC training and their comfort level. Comfort level with some clinical situations was also significantly different based on year of training. Fellowship training programs should have CCC curricula which use experiential learning models and lay the foundation for promoting cultural awareness, self-reflection, and better patient-physician partnerships which can eventually adapt to and surmount the challenges unique to the physician's chosen field of practice.
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Diversidade Cultural , Atenção à Saúde/normas , Docentes de Medicina/normas , Bolsas de Estudo/normas , Hematologia/educação , Oncologia/educação , Pediatria/educação , Adulto , Criança , Comunicação , Currículo , Educação de Pós-Graduação em Medicina , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Relações Médico-Paciente , Inquéritos e QuestionáriosRESUMO
Rates of venous thromboembolism have increased in the adolescent population over the last two decades, likely due to advanced diagnostics, increased use of central venous catheters, chronic medical conditions, obesity, and oral contraceptive use. Of these factors, a modifiable risk factor for adolescents is obesity. Sedentary lifestyle and prolonged immobilization are additional prothrombotic risk factors that are often associated with obesity. With ever-increasing screen time, sedentary behavior has risen accordingly, especially among gamers. We present four cases of adolescents who developed life-threatening venous thromboembolic events in the setting of obesity, sedentary lifestyle and/or immobilization, and prolonged video game use.
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Obesidade/complicações , Comportamento Sedentário , Trombofilia/etiologia , Tromboembolia Venosa/etiologia , Jogos de Vídeo/efeitos adversos , Adolescente , Comportamento do Adolescente , Humanos , MasculinoRESUMO
Late-onset HC is a well-recognized complication associated with cyclophosphamide/acrolein-induced toxicity. It poses a management challenge when hyperhydration and bladder irrigation do not result in clinical improvement as desired. The data regarding use of hyperbaric oxygen therapy (HBO2) as an early treatment modality in this clinical setting are limited. We present 2 cases, that were refractory to hyperhydration and bladder irrigation but responded to HBO2. They were treated with 20-30 daily sessions over weekdays with 100% oxygen for 90 minutes at 2 atmospheric pressure units (2 atm). Both patients reported improved symptoms within the first 15 sessions, and hematuria diminished by 20 sessions. Hyperbaric oxygen is a less invasive, outpatient therapy that is effective for treatment of HC and is tolerated well by young patients.
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Ciclofosfamida/efeitos adversos , Cistite/terapia , Hematúria/terapia , Oxigenoterapia Hiperbárica , Imunossupressores/efeitos adversos , Adolescente , Adulto , Cistite/induzido quimicamente , Feminino , Hematúria/induzido quimicamente , Humanos , MasculinoRESUMO
HSTCL is a highly aggressive malignancy with a poor prognosis. Case series and accounts have reported the use of different chemotherapy regimens with diverse patient outcomes. Most long-term survivors had undergone high-dose chemotherapy with autologous or allogeneic HCT. We describe two pediatric patients with HSTCL who were treated with chemotherapy followed by allogeneic HCT. Both patients are alive and in complete remission 2 and 8 years after therapy. Multiagent chemotherapy followed with allogeneic HCT seems to provide patients who have chemotherapy-sensitive disease a long-term disease-free survival.
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Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas , Neoplasias Hepáticas/terapia , Linfoma de Células T/terapia , Neoplasias Esplênicas/terapia , Adolescente , Criança , Terapia Combinada , Humanos , Adulto JovemRESUMO
The risk of viral infections and reactivation occurring in the setting of pediatric allogeneic hematopoietic stem cell transplantation is a concern in the pediatric patient, especially with the use of Alemtuzumab (Campath) as a conditioning agent. The purpose of this study was to determine the incidence of Epstein-Barr virus posttransplant lymphoproliferative disorder (EBV-PTLD), cytomegalovirus (CMV), and adenovirus among pediatric recipients of alemtuzumab at our institution. We found that EBV-PTLD occurred in 2.1% of transplants (1 matched unrelated donor [MUD] recipient), CMV reactivation occurred in 12.5% of transplants (4 MUD and 2 matched related donor [MRD] recipients) with disseminated CMV in 2.1% of cases (1 MRD recipient), and adenovirus infection occurred in 8.3% of the total transplants (2 MUD and 2 MRD recipients). Alemtuzumab continues to be used as a method of graft-versus-host disease and graft failure prevention among pediatric recipients of hematopoietic stem cell transplantation and seems to be safer than previously reported. At our institution, alemtuzumab has not increased the risk for EBV-PTLD, CMV infection, or adenovirus.
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Infecções por Adenoviridae/epidemiologia , Alemtuzumab/administração & dosagem , Infecções por Citomegalovirus/epidemiologia , Infecções por Vírus Epstein-Barr/epidemiologia , Transtornos Linfoproliferativos/epidemiologia , Condicionamento Pré-Transplante , Doadores não Relacionados , Adenoviridae , Infecções por Adenoviridae/etiologia , Adolescente , Adulto , Alemtuzumab/efeitos adversos , Aloenxertos , Criança , Pré-Escolar , Citomegalovirus , Infecções por Citomegalovirus/etiologia , Infecções por Vírus Epstein-Barr/etiologia , Feminino , Seguimentos , Rejeição de Enxerto/epidemiologia , Rejeição de Enxerto/prevenção & controle , Doença Enxerto-Hospedeiro/epidemiologia , Doença Enxerto-Hospedeiro/prevenção & controle , Herpesvirus Humano 4 , Humanos , Lactente , Recém-Nascido , Transtornos Linfoproliferativos/etiologia , Masculino , Estudos Retrospectivos , Fatores de RiscoRESUMO
We report a 9-year-old Chinese girl with congenital thrombotic thrombocytopenic purpura found to be a compound heterozygote for 2 pathogenic variants in the ADAMTS13 gene, including a novel variation. The girl suffered from recurrent, life-threatening episodes of thrombocytopenia and hemolysis, and laboratory testing showed ADAMST13 enzyme activity of <5%. Sequencing of the ADAMTS13 gene revealed a previously reported missense variant, c.1787C>T (p.Ala596Val), and a novel duplication defined as c.1007_1025dup19 (p.Asp343Leufs*53); the duplication is predicted to result in a premature stop codon and protein truncation. We propose that this novel variant is partly responsible for the patient's early-onset and severe phenotype.
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Proteína ADAMTS13/genética , Códon sem Sentido , Mutação de Sentido Incorreto , Púrpura Trombocitopênica Trombótica/genética , Proteína ADAMTS13/deficiência , Idade de Início , Povo Asiático , Criança , Feminino , Hemólise , Heterozigoto , Humanos , Púrpura Trombocitopênica Trombótica/patologia , TrombocitopeniaRESUMO
High-dose methotrexate has been a treatment for osteosarcoma; however, its nephrotoxic effects are considerable. Carboxypeptidase-G2 (glucarpidase) was approved by the US Food and Drug Administration in 2012 for treatment of toxic methotrexate levels. We report our experience using glucarpidase under compassionate use before Food and Drug Administration approval in 2 patients who had delayed methotrexate clearance and prolonged kidney injury despite glucarpidase administration. Our results show that patients with methotrexate toxicity may require repeated doses of glucarpidase in addition to supportive measures, such as dialysis.