RESUMO
A 9-year-old boy with acute lymphoblastic leukemia experienced a hypersensitivity reaction (HSR) and acral erythema upon receiving high-dose methotrexate (HDMTX). Both HSR and acral erythema are uncommon adverse events of MTX therapy, and MTX has not been reported to cause HSRs in specific ethnic groups. We assessed the severity of each symptom and were successful in managing these adverse events for continuing subsequent HDMTX therapies. HSR appeared during the first and second HDMTX courses. Acral erythema occurred after the second and fourth courses. Desensitization by reducing the infusion rate and premedication allowed the continuation of HDMTX. Acral erythema improved with supportive care without dose reduction or interval lengthening. HSRs to MTX should be considered even during the first course. MTX-induced acral erythema is a self-limited reaction; therefore, the chemotherapeutic regimen should not be modified unless necessary.
Assuntos
Hipersensibilidade , Leucemia-Linfoma Linfoblástico de Células Precursoras , Antimetabólitos Antineoplásicos/uso terapêutico , Criança , Eritema/induzido quimicamente , Eritema/diagnóstico , Humanos , Masculino , Metotrexato , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológicoRESUMO
BACKGROUND: Cervical lymphadenitis (CL) cannot be easily distinguished from Kawasaki disease (KD). We therefore explored whether brain natriuretic peptide (BNP) levels are useful in this context. METHODS: We retrospectively analyzed 14 children with CL and 177 children with KD. Patients with KD were divided into three groups according to their clinical symptoms at hospitalization - 97 patients had typical KD, 35 had node-first KD (NFKD), and 45 had KD without lymphadenopathy. We reviewed data on clinical and laboratory parameters, including serum BNP levels, at hospitalization together with factors that might distinguish KD from CL. RESULTS: Patients with CL were older than those with KD. Serum BNP levels were higher in all the KD groups than in the CL group. Multivariate logistic regression analyses indicated that higher BNP levels were associated with NFKD (odds ratio: 1.12, 95% confidence interval: 1.01-1.25). The receiver operating characteristic curve yielded a BNP cutoff of 18.3 pg/mL, with a sensitivity of 0.680, a specificity of 0.857, and an area under the curve of 0.806 (95% confidence interval: 0.665-0.947). CONCLUSIONS: Serum BNP levels can be used to distinguish KD from CL, especially in patients with NFKD.
Assuntos
Linfadenite , Síndrome de Linfonodos Mucocutâneos , Criança , Humanos , Síndrome de Linfonodos Mucocutâneos/diagnóstico , Peptídeo Natriurético Encefálico , Estudos Retrospectivos , Linfadenite/diagnóstico , Curva ROC , Biomarcadores , Fragmentos de PeptídeosRESUMO
A 1-year-old boy presented with a 4-month history of hypertension, ptosis of the right upper eyelid, left hemifacial sweating, and flushing. He was diagnosed with Harlequin syndrome associated with Horner syndrome. Computed tomography revealed a mass lesion in the right superior mediastinum. Therefore, the patient underwent total tumor resection. Histological examination demonstrated ganglioneuroblastoma. The MYCN oncogene was not amplified, and the mitosis-karyorrhexis index was low. Accordingly, radiation and chemotherapy were not performed. No recurrence was observed within 8 months after surgery, and the patient's blood pressure was normalized. However, the ptosis, hemifacial sweating, and flushing persisted.
Assuntos
Ganglioneuroblastoma , Síndrome de Horner , Doenças do Sistema Nervoso Autônomo , Rubor/etiologia , Ganglioneuroblastoma/complicações , Ganglioneuroblastoma/diagnóstico por imagem , Ganglioneuroblastoma/cirurgia , Síndrome de Horner/etiologia , Humanos , Hipo-Hidrose , Lactente , Masculino , Recidiva Local de NeoplasiaRESUMO
Glucocorticoid (GC) resistance is associated with poor response to the following chemotherapy in lymphoid malignancies, such as lymphoma and leukemia. However, it remains unclear whether GCs interfere with the cytotoxic effects of anti-cancer drugs on GC-resistant cells. In this study, we examined whether GCs affected the sensitivities to vincristine (VCR)/doxorubicin (DOX) and the expression of drug transporters in GC-resistant cells. The dexamethasone (DEX)/prednisolone (PSL)-resistant lymphoid and non-lymphoid cell lines Raji and HL60 were cultured with DEX for 7 days and then treated with VCR or DOX for 3 days. Seven days of DEX treatment increased the IC50s of both VCR and DOX in Raji cells but not in HL60 cells. The mRNA and protein expression levels of organic cation/carnitine transporter (OCTN) 2, one of the drug uptake transporters expressed in both cell lines, were decreased only in Raji cells. When Raji cells were cultured with PSL, the IC50 of DOX but not VCR increased as the expression of OCTN2 decreased. No significant increases in efflux transporter expression were induced by DEX or PSL. When siRNA against OCTN2 was introduced into Raji cells, the IC50 of DOX but not VCR increased significantly. These data suggested that both DEX and PSL decreased the sensitivity of the DEX/PSL-resistant Raji cells to DOX, a change that was at least partially due to reductions in OCTN2. Thus, the continuous usage of GCs may interfere with the effects of chemotherapy on GC-resistant lymphoid cells.
Assuntos
Dexametasona/farmacologia , Doxorrubicina/farmacologia , Resistencia a Medicamentos Antineoplásicos/efeitos dos fármacos , Glucocorticoides/farmacologia , Linfócitos/metabolismo , Proteínas de Neoplasias/metabolismo , Prednisolona/farmacologia , Membro 5 da Família 22 de Carreadores de Soluto/metabolismo , Células HL-60 , Humanos , Leucemia/tratamento farmacológico , Leucemia/metabolismo , Leucemia/patologia , Linfócitos/patologia , Linfoma/tratamento farmacológico , Linfoma/metabolismo , Linfoma/patologiaRESUMO
Intensive chemotherapy with tyrosine kinase inhibitor (TKI) improves the prognosis of patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph-ALL). However, the prognosis of cases of relapsed or refractory Ph-ALL remains poor. Here, we aimed to assess the efficacy of T-cell-rich HLA-haploidentical hematopoietic stem cell transplantation (TCR-haplo-HSCT) in eight patients with relapsed or refractory pediatric Ph-ALL. Transplant-related mortality was observed in two patients. All patients discontinued TKI after receiving TCR-haplo-HSCT. The 3-year probability of overall survival and event-free survival was 75.0 and 62.5%, respectively. These results indicate the efficacy of TCR-haplo-HSCT for relapsed/refractory pediatric Ph-ALL.
Assuntos
Resistencia a Medicamentos Antineoplásicos , Antígenos HLA/imunologia , Transplante de Células-Tronco Hematopoéticas , Recidiva Local de Neoplasia/terapia , Cromossomo Filadélfia , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Linfócitos T/imunologia , Adolescente , Criança , Pré-Escolar , Feminino , Seguimentos , Humanos , Lactente , Masculino , Recidiva Local de Neoplasia/imunologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/imunologia , Prognóstico , Inibidores de Proteínas Quinases/uso terapêutico , Proteínas Tirosina Quinases/antagonistas & inibidores , Estudos Retrospectivos , Terapia de Salvação , Condicionamento Pré-Transplante , Transplante HomólogoRESUMO
GF is one of the fatal complications of allogeneic HSCT. To rescue patients with primary GF, a second HSCT should be conducted as soon as possible, but the optimal donor source and technique have yet to be established. In this study, we retrospectively analyzed six children with hematologic malignancies who received TCR-haploidentical second HSCT for primary GF. The median interval between the prior HSCT and the second HSCT was 37.5 days. All patients received fludarabine and ATG containing reduced-intensity re-conditioning before the second HSCT. All patients, except one who died early, achieved both neutrophil and Plt engraftment at a median time of 15 and 33 days, respectively. Chimerism analysis showed that all engrafted patients achieved complete donor chimerism within 3 weeks. Four patients developed acute GVHD, and three patients developed chronic GVHD. TRM occurred in two patients. Median follow-up of the four survivors was 6.8 years, and all remained in sustained remission until the last follow-up. These results suggested that a TCR-haploidentical second HSCT for pediatric patients is feasible, and this approach may provide a potent option for children with primary GF.
Assuntos
Neoplasias Hematológicas/terapia , Transplante de Células-Tronco Hematopoéticas/métodos , Linfócitos T/transplante , Transplante Haploidêntico/métodos , Adolescente , Criança , Seguimentos , Sobrevivência de Enxerto , Humanos , Estudos Retrospectivos , Condicionamento Pré-Transplante/métodos , Resultado do TratamentoRESUMO
BACKGROUND: Pediatric apheresis for peripheral blood stem cell transplantation should be carried out with due concern for low corporeal blood volume and vulnerability to hypocalcemia-related complications, hypovolemic shock, and hypervolemic cardiac overload. STUDY DESIGN AND METHODS: We retrospectively investigated a total of 267 apheresis procedures from 1990 to 2013 on 93 children between 0 and 10 years old, including 89 patients and 4 healthy donors, with body weights of 6.3 to 44.0 kg. RESULTS: The median CD34+ cell yield per apheresis procedure was 2.3 × 106 CD34+ cells/kg (0.2-77.9 × 106 CD34+ cells/kg). Adverse events occurred in 11.6% of procedures (n = 31), including mild perivascular pain (n = 12), emesis (n = 9), hypotension (n = 3), urticaria (n = 2), numbness (n = 2), chest pain (n = 1), facial flush (n = 1), and abdominal pain (n = 1). Among hypotensive events, shock in a 9.6 kg one-year-old boy required emergency treatment in 1996. Thereafter, we adopted continuous injection of calcium gluconate, ionized calcium monitoring, central venous catheter access and circuit priming with albumin in addition to concentrated red cells. Since then we have had fewer complications: 16.4% per apheresis during 1990-1997 versus 5.8% during 1998-2013. No healthy pediatric donors suffered from any late-onset complications related to apheresis or G-CSF administration. CONCLUSION: By employing appropriate measures, peripheral blood stem cell apheresis for small children can have an improved safety profile, even for children weighing <10 kg.
Assuntos
Remoção de Componentes Sanguíneos/métodos , Mobilização de Células-Tronco Hematopoéticas/métodos , Assistência ao Paciente/métodos , Células-Tronco de Sangue Periférico/citologia , Antígenos CD34/metabolismo , Remoção de Componentes Sanguíneos/efeitos adversos , Doadores de Sangue , Pressão Sanguínea , Peso Corporal/efeitos dos fármacos , Cálcio/administração & dosagem , Cálcio/farmacologia , Criança , Pré-Escolar , Suplementos Nutricionais , Feminino , Humanos , Lactente , Recém-Nascido , Masculino , Dor/etiologia , Células-Tronco de Sangue Periférico/efeitos dos fármacosRESUMO
Idiopathic hyperammonemia (IHA) has been described as a complication of intensive chemotherapy for the treatment of hematologic malignancy but has subsequently been found in patients undergoing bone marrow transplantation and in those with solid tumors treated with 5-fluorouracil. Although IHA is a rare complication, it is sometimes associated with high mortality in hematologic malignancies. Here we report the case of a 15-year-old boy in whom hyperammonemia developed during the initial treatment with prednisolone for newly diagnosed acute lymphoblastic leukemia and who survived after early detection and oral lactulose therapy. To the best of our knowledge, this is the first report of IHA that was not induced by intensive chemotherapy, stem cell transplantation, or asparaginase therapy in a patient with newly diagnosed leukemia, but developed during an initial treatment with a steroid. Early detection of IHA by measuring the plasma ammonia level in patients with neurological symptoms may improve the outcome.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Hiperamonemia/induzido quimicamente , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Adolescente , Citarabina/administração & dosagem , Humanos , Hidrocortisona/administração & dosagem , Hiperamonemia/tratamento farmacológico , Hiperamonemia/patologia , Lactulose/uso terapêutico , Masculino , Metotrexato/administração & dosagem , Leucemia-Linfoma Linfoblástico de Células Precursoras/diagnóstico , Prednisolona/administração & dosagem , PrognósticoRESUMO
Chronic active Epstein-Barr virus (EBV) infection (CAEBV), characterized by persistent infectious mononucleosis-like symptoms, can lead to cardiovascular complications including coronary artery aneurysm or myocarditis. Here, we present the case of an 11-year-old boy with pulmonary arterial hypertension (PAH) and junctional ectopic tachycardia associated with CAEBV. The patient did not have any major symptoms attributed to CAEBV, such as fever, lymphadenopathy or splenomegaly when the PAH developed. Mild liver dysfunction was found at the first examination, and it persisted. Two years after the PAH symptoms appeared, CAEBV was evident, based on deteriorated liver function, hepatosplenomegaly, and coronary artery aneurysms. CAEBV should be considered as a cause of secondary PAH, particularly when liver dysfunction coexists.
Assuntos
Infecções por Vírus Epstein-Barr/virologia , Hipertensão Pulmonar/virologia , Taquicardia Ectópica de Junção/virologia , Criança , Doença Crônica , Ecocardiografia , Eletrocardiografia , Infecções por Vírus Epstein-Barr/diagnóstico por imagem , Evolução Fatal , Humanos , Hipertensão Pulmonar/diagnóstico por imagem , Masculino , Transplante de Células-Tronco de Sangue Periférico , Taquicardia Ectópica de Junção/diagnóstico por imagem , Transplante HomólogoRESUMO
Malignant peritoneal mesothelioma in children is a very rare disease and has a poor prognosis. Unlike malignant mesothelioma in adults, there is no clear causal association between this very rare malignancy in children and asbestos exposure. We report a case of peritoneal mesothelioma in an 11-year-old boy who presented with ascites. He was diagnosed with malignant mesothelioma on the basis of histopathological findings. His disease showed resistance to pemetrexed, but was treated successfully with platinum-based therapy with gemcitabine. He has achieved long-term survival in partial remission with stable disease.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Neoplasias Pulmonares/tratamento farmacológico , Mesotelioma/tratamento farmacológico , Neoplasias Peritoneais/tratamento farmacológico , Ascite/tratamento farmacológico , Criança , Cisplatino/administração & dosagem , Desoxicitidina/administração & dosagem , Desoxicitidina/análogos & derivados , Resistencia a Medicamentos Antineoplásicos , Glutamatos/uso terapêutico , Guanina/análogos & derivados , Guanina/uso terapêutico , Humanos , Masculino , Mesotelioma Maligno , Pemetrexede , GencitabinaRESUMO
Myeloid/natural killer cell precursor acute leukemia (MNKL) is an aggressive disease with a high relapse rate even after allogeneic hematopoietic stem cell transplantation (SCT). We report a patient with MNKL who had a donor lymphocyte infusion (DLI) for relapse after T cell-replete human leukocyte antigen (HLA)-haploidentical SCT, but relapsed again 20 months later with loss of mismatched HLA. This case suggests that a strong graft-versus-leukemia effect of haploidentical SCT can be expected in MNKL patients. In the haploidentical setting, DLI should be considered for patients with relapsed leukemia whose leukemic cells have not lost HLA cell surface expression.
Assuntos
Antígenos HLA/imunologia , Transplante de Células-Tronco Hematopoéticas , Leucemia Mieloide Aguda/imunologia , Recidiva Local de Neoplasia/imunologia , Adolescente , Doença Enxerto-Hospedeiro/imunologia , Humanos , Células Matadoras Naturais/patologia , Leucemia Mieloide Aguda/cirurgia , MasculinoRESUMO
BACKGROUND: The objective of this study was to evaluate the efficacy and safety of our combination therapy in febrile neutropenic children with cancer. METHODS: A total of 109 patients with 251 episodes of febrile neutropenia received antibiotic therapy between January 2003 and December 2008 at a single institution. RESULTS: Blood cultures were positive in 35 episodes (14%). Gram-positive organisms predominated (23/38 organisms isolated). There were 15 gram-negative isolates and no fungal isolates. The recommended empirical first-line antibiotics (cefepime or cefozopran + piperacillin + amikacin) were used in 206 (82%), second-line antibiotics (piperacillin-tazobactam + carbapenem + amikacin + micafungin) in 73 (29%), and third-line antibiotics (meropenem + glycopeptides + micafungin) in 24 (10%) episodes. The overall response rates were 71.4%, 50.7%, and 62.5% for the first-, second-, and third-line antibiotic therapies, respectively. Granulocyte transfusion was performed in seven patients, and the response rate was 57%. Four deaths were recorded. CONCLUSIONS: Although a significant improvement of mortality was not observed, our regimen of empirical antibiotic therapies led to a significant and clinically relevant decrease in glycopeptide use, and it is safe and well tolerated by pediatric neutropenic patients.
Assuntos
Antibacterianos/uso terapêutico , Neutropenia Febril Induzida por Quimioterapia/terapia , Infecções por Bactérias Gram-Negativas/terapia , Infecções por Bactérias Gram-Positivas/terapia , Granulócitos , Transfusão de Leucócitos , Adolescente , Algoritmos , Antineoplásicos/efeitos adversos , Neutropenia Febril Induzida por Quimioterapia/diagnóstico , Neutropenia Febril Induzida por Quimioterapia/mortalidade , Criança , Pré-Escolar , Terapia Combinada , Técnicas de Apoio para a Decisão , Quimioterapia Combinada , Feminino , Infecções por Bactérias Gram-Negativas/diagnóstico , Infecções por Bactérias Gram-Negativas/mortalidade , Infecções por Bactérias Gram-Positivas/diagnóstico , Infecções por Bactérias Gram-Positivas/mortalidade , Transplante de Células-Tronco Hematopoéticas , Humanos , Lactente , Recém-Nascido , Masculino , Neoplasias/complicações , Neoplasias/tratamento farmacológico , Neoplasias/cirurgia , Condicionamento Pré-Transplante/efeitos adversos , Resultado do Tratamento , Adulto JovemRESUMO
BACKGROUND: Murine double minute 2 (MDM2) is an oncogene that inhibits p53, leading to decreased apoptosis. Sarcomas showing MDM2 amplification are rare among pediatric patients. CASE: A 14-year-old boy presented with pleomorphic sarcoma of the head showing MDM2 amplification without a well-differentiated liposarcoma component. Although chemotherapy was initially performed to reduce the tumor size before surgery, the tumor did not shrink. The patient underwent complete surgical resection. Microscopic examination revealed a positive surgical margin; thus, postoperative proton-beam radiotherapy was performed. 3 years after the therapy, no sign of recurrence was observed. CONCLUSION: Macroscopic surgical resection combined with adjuvant postoperative radiotherapy was effective against MDM2-amplified pleomorphic sarcoma refractory to neoadjuvant chemotherapy in a pediatric patient.
Assuntos
Lipossarcoma , Sarcoma , Neoplasias de Tecidos Moles , Masculino , Humanos , Criança , Animais , Camundongos , Adolescente , Proteínas Proto-Oncogênicas c-mdm2/genética , Proteínas Proto-Oncogênicas c-mdm2/metabolismo , Hibridização in Situ Fluorescente , Amplificação de Genes , Lipossarcoma/diagnóstico , Lipossarcoma/genética , Lipossarcoma/patologia , Sarcoma/patologia , Neoplasias de Tecidos Moles/diagnósticoRESUMO
Western countries that were first to administer the COVID-19 vaccination report cases of vaccine-induced axillary lymphadenitis with high FDG uptake. However, no such findings have been reported from any Asian countries. We report here a confusing case of a 31-year-old female cancer survivor with high FDG uptake in her axillary lymph nodes, suggesting recurrence, following mRNA COVID-19 vaccination. Although the value of SUVmax was elevated (12.7), additional imaging revealed that her lymphatic lesions were benign, and they resolved spontaneously. This case of a strong immune reaction to COVID-19 vaccination in regional lymph nodes is the first reported in a Japanese patient. We should be aware of this new mimic and optimize diagnostic imaging methods accordingly in the era of COVID-19.
Assuntos
COVID-19 , Sobreviventes de Câncer , Linfadenite , Adulto , Vacinas contra COVID-19 , Feminino , Fluordesoxiglucose F18 , Humanos , Linfadenite/induzido quimicamente , Linfadenite/diagnóstico , Metástase Linfática , Recidiva Local de Neoplasia , RNA Mensageiro , SARS-CoV-2RESUMO
In this study, we evaluated the feasibility of our graft-versus-host disease (GVHD) prophylaxis with tacrolimus, methotrexate, and prednisolone in non-T-cell-depleted haploidentical hematopoietic stem cell transplantation (HSCT) for children. Twenty-one consecutive patients including those with hematological malignancies (n = 11), solid tumors (n = 7), and non-malignancies (n = 3) were analyzed. Myeloablative and reduced intensity conditionings were carried out in 5 and 16 patients, respectively, and both of the regimens contained anti-human T-lymphocyte immunoglobulin. Twenty (95%) of the 21 patients achieved primary engraftment. Acute GVHD of grades II-IV and III-IV were observed in nine (47%) and one (5%) patient, respectively, all of which were controllable by steroids. Chronic GVHD was observed in eight (51%) of the 17 evaluable patients, and one of them developed steroid refractory chronic GVHD. Treatment-related mortality occurred in three patients (15%), as a result of acute pancreatitis, chronic GVHD, and EB virus associated lymphoproliferative disease. The median follow-up of the 13 survivors was 24 months, and the two-yr probability of overall survival was 68%. The Karnofsky performance scale score of the 13 survivors was 100%. These results indicated the feasibility of our GVHD prophylaxis in non-T-cell-depleted haploidentical HSCT for children.
Assuntos
Doença Enxerto-Hospedeiro/prevenção & controle , Neoplasias Hematológicas/terapia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Metotrexato/uso terapêutico , Prednisolona/uso terapêutico , Linfócitos T/imunologia , Tacrolimo/uso terapêutico , Adolescente , Adulto , Anti-Inflamatórios/uso terapêutico , Criança , Pré-Escolar , Estudos de Viabilidade , Feminino , Seguimentos , Doença Enxerto-Hospedeiro/epidemiologia , Doença Enxerto-Hospedeiro/etiologia , Neoplasias Hematológicas/imunologia , Histocompatibilidade , Humanos , Imunossupressores/uso terapêutico , Incidência , Lactente , Recém-Nascido , Japão/epidemiologia , Masculino , Prognóstico , Taxa de Sobrevida , Transplante Homólogo , Adulto JovemRESUMO
Because the Bacillus Calmette-Guérin (BCG) prevents infants from contracting miliary tuberculosis and tuberculosis meningitis, BCG vaccination is recommended for those under 6 months old in Japan. Complications such as favorable local inflammatory reactions including redness, induration, and abscess formation may occur, but severe adverse effects such as osteomyelitis, periostitis, and disseminated BCG infection are generally rare. We report an 11-month-old boy with severe combined immunodeficiency dying of serious disseminated BCG infection despite anti-tuberculosis therapy and blood stem cell transplantation. He was vaccinated with disseminated BCG infection at 4 months before severe combined immunodeficiency diagnosis was confirmed by specific RD gene deletion based on allele-specific polymerase chain reaction. Although BCG is considered safe, we should keep in mind that subjects with immunological deficiency may suffer severe BCG complications.
Assuntos
Vacina BCG/efeitos adversos , Deleção de Genes , Mycobacterium bovis/genética , Imunodeficiência Combinada Severa/complicações , Tuberculose/etiologia , Humanos , Lactente , Masculino , Vacinação/efeitos adversosRESUMO
OBJECTIVE: To investigate the usefulness of procalcitonin (PCT) as predictive factors of intravenous immunoglobulin (IVIG)-resistant Kawasaki disease patients. METHODS: We retrospectively analyzed the laboratory data from 215 children with Kawasaki disease treated with IVIG from 2014 to 2019. We analyzed the clinical and laboratory parameters just before the IVIG including serum levels of PCT with respect to the IVIG response. RESULTS: Eventually, 127 patients were analyzed. The median age was 2.4 years. IVIG was effective in 108 children (responders) and was ineffective in 19 (non-responders). Serum PCT concentration was higher in non-responders than those of responders (P < 0.001). Multivariate logistic regression analyses indicated that higher PCT concentration (odds ratio 1.34, 95% confidence interval 1.10-1.64) were associated with IVIG resistance. Analyses of the receiver operating characteristic curve showed that the cutoff value of PCT 2.18 ng/mL had 46.4% of sensitivity and 93.9% of specificity. Receiver operating characteristic analysis yielded an area under the curve of 0.82 (0.72-0.92) to predict IVIG resistance. CONCLUSIONS: Serum PCT value can be an excellent biomarker for predicting unresponsiveness to IVIG with a good discriminatory ability as well as the existing prediction scores.
Assuntos
Imunoglobulinas Intravenosas/uso terapêutico , Síndrome de Linfonodos Mucocutâneos/terapia , Pró-Calcitonina/sangue , Biomarcadores/sangue , Criança , Pré-Escolar , Feminino , Humanos , Imunoglobulinas Intravenosas/normas , Lactente , Modelos Logísticos , Masculino , Síndrome de Linfonodos Mucocutâneos/diagnóstico , Análise Multivariada , Razão de Chances , Valor Preditivo dos Testes , Pró-Calcitonina/normas , Estudos Retrospectivos , Falha de TratamentoRESUMO
CGD is a rare inherited immunodeficiency disorder that is caused by disability of oxidative killing. We presented a two-yr-old boy with CGD who was suffering from multiple systemic abscesses. He received the first BMT from his HLA-haploidentical mother after conditioning with Flu, melphalan, and ATG. Although the maximum of 42% donor chimerism was achieved, it disappeared 73 days after the BMT. Then, we performed 5/6-matched unrelated cord blood re-transplantation after conditioning with Flu, Bu, and TBI (2 Gy). Engraftment and complete donor chimerism were achieved on days 18 and 19, respectively. The patient is now free from infection and maintains complete donor chimerism without GVHD 36 months after the cord blood re-transplantation. We postulate that the unrelated CBT has a potential to be an alternative strategy and might be beneficial for patients with CGD who do not have an HLA-identical donor.