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Atsushi Narita,
Hideki Muramat,
Hirotoshi Sakaguchi,
Sayoko Doisaki,
Makito Tanaka,
Asahito Hama,
Akira Shimada,
Yoshiyuki Takahashi,
Nao Yoshida, Kimikazu Matsumoto,
Koji Kato,
Kazuko Kudo,
Yoko Furukawa-Hibi,
Kiyofumi Yamada,
Seiji Kojima
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ABSTRACT: BACKGROUND:: Voriconazole is a triazole antifungal agent with potent activity against a broad spectrum of pathogens, including Aspergillus and Candida species. In human adults, allelic polymorphisms of CYP2C19 are known to correlate with significant variation in voriconazole plasma concentration. Here, we report an analysis of CYP2C19 phenotype and voriconazole plasma concentrations in children. METHODS:: This retrospective study included 37 children who had voriconazole plasma concentrations measured from May 2006 to June 2011. All had single-nucleotide polymorphisms that define the 3 major CYP2C19 alleles. Patients were classified as follows: normal metabolizers, intermediate metabolizers, poor metabolizers, or hypermetabolizers. RESULTS:: The frequencies of the 3 CYP2C19 genetic polymorphisms were similar to those previously reported for Japanese adults. Trough plasma concentrations of voriconazole were significantly higher in the poor metabolizer and intermediate metabolizer groups compared with the normal metabolizer and hypermetabolizer groups (P=0.004). Two patients with high plasma concentrations experienced voriconazole-related severe adverse events (syndrome of inappropriate antidiuretic hormone secretion and cardiac toxicities). CONCLUSIONS:: The current study suggests that a significant association exists in children between the voriconazole plasma concentration and the CYP2C19 phenotype. Dose adjustment based on CYP2C19 phenotype may be useful during voriconazole therapy, especially for Japanese children, who as a group have a higher incidence of the poor metabolizer and intermediate metabolizer phenotypes.
Journal of Pediatric Hematology/Oncology 04/2013; · 1.16 Impact Factor
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ABSTRACT: Pulmonary hypertension (PH) is an infrequently reported complication after hematopoietic stem cell transplantation, and its etiology and therapeutic strategies, especially in infants, remain unclear. We report a case of severe PH that developed in an infant with acute leukemia following administration of busulfan as a preconditioner for cord blood transplantation; the case was successfully treated with sildenafil and beraprost, which to our knowledge is the first reported successful use of this regimen in PH following transplantation for infantile leukemia. From a review of all previous reports, use of busulfan in infants may raise the risk of developing PH, and unlike definitive pulmonary veno-occlusive disease, PH in this subgroup may be reversible by early detection and treatment.
International journal of hematology 12/2012; · 1.17 Impact Factor
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Masaru Ihira,
Yoshihiko Enomoto,
Yoshiki Kawamura,
Hidetaka Nakai,
Ken Sugata,
Yoshizo Asano,
Motohiro Tsuzuki,
Nobuhiko Emi,
Tatsunori Goto,
Koichi Miyamura, Kimikazu Matsumoto,
Koji Kato,
Yoshiyuki Takahashi,
Seiji Kojima,
Tetsushi Yoshikawa
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ABSTRACT: The monitoring of active human herpesvirus 6 (HHV-6) B infection is important for distinguishing between the reactivation and latent state of the virus. The aim of this present study is to develop a quantitative reverse transcription polymerase chain reaction (RT-PCR) assay for diagnosis of active viral infection. Primers and probes for in house quantitative RT-PCR methods were designed to detect the three kinetic classes of HHV-6B mRNAs (U90, U12, U100). Stored PBMCs samples collected from 10 patients with exanthem subitum (primary HHV-6B infection) and 15 hematopoietic stem cell transplant recipients with HHV-6B reactivation were used to evaluate reliability for testing clinical samples. Excellent linearity was obtained with high correlation efficiency between the diluted RNA (1-100 ng/reaction) and C(t) value of each gene transcript. The U90 and U12 gene transcripts were detected in all of the peripheral blood mononuclear cells (PBMCs) samples collected in acute period of primary HHV-6B infection. Only one convalescent PBMCs sample was positive for the U90 gene transcript. Additionally, the reliability of HHV-6B quantitative RT-PCRs for diagnosis of viral reactivation in hematopoietic transplant recipients was evaluated. Relative to virus culture, U90 quantitative RT-PCR demonstrated the highest assay sensitivity, specificity, positive predictive value, and negative predictive value. Thus, this method could be a rapid and lower cost alternative to virus culture, which is difficult to perform generally, for identifying active HHV-6B infection.
Journal of Medical Virology 09/2012; 84(9):1388-95. · 2.82 Impact Factor
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ABSTRACT: Hemorrhagic cystitis is critical in patients with hemato-oncological disorders. Unlike adult patients, there are limited modalities and invasive procedures are often not well tolerated in children with poor general conditions. We report a pediatric patient with refractory acute lymphoblastic leukemia who developed life-threatening massive gross hematuria. Along with platelet infusion every other day due to suppressed hematopoiesis, his gross hematuria and clot retention in the bladder were successfully treated with choreito, a formula from Japanese traditional medicine (Kampo medicine). He survived free from hematuria for more than four months. Choreito was well tolerated, and no adverse effects were observed throughout the course.
Phytomedicine: international journal of phytotherapy and phytopharmacology 07/2012; 19(12):1143-6. · 2.17 Impact Factor
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British Journal of Haematology 06/2012; 158(5):666-8. · 4.94 Impact Factor
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The Journal of infection 06/2012; 65(4):366-7. · 4.13 Impact Factor
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Akira Shimada,
Yoshiyuki Takahashi,
Hideki Muramatsu,
Asahito Hama,
Olfat Ismael,
Atsushi Narita,
Hiroshi Sakaguchi,
Sayoko Doisaki,
Nobuhiro Nishio,
Makito Tanaka,
Nao Yoshida, Kimikazu Matsumoto,
Koji Kato,
Nobuhiro Watanabe,
Seiji Kojima
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ABSTRACT: Fanconi anemia (FA) is a disorder characterized by developmental anomalies, bone marrow failure and a predisposition to malignancy. It has recently been shown that hematopoietic stem cell transplantation using fludarabine (FLU)-based reduced-intensity conditioning is an efficient and quite safe therapeutic modality. We retrospectively analyzed the outcome of bone marrow transplantation (BMT) in eight patients with FA performed in two institutes between 2001 and 2011. There were seven females and one male with a median age at diagnosis = 4.5 years (range 2-12 years). The constitutional characteristics associated with FA, such as developmental anomalies, short stature and skin pigmentation, were absent in three of the patients. One patient showed myelodysplastic features at the time of BMT. All patients received BMT using FLU, cyclophosphamide (CY) and rabbit anti-thymocyte globulin (ATG) either from a related donor (n = 4) or an unrelated donor (n = 4). Acute graft-versus-host disease (GVHD) of grade I developed in one patient, while chronic GVHD was not observed in any patient. All patients are alive and achieved hematopoietic recovery at a median follow-up of 72 months (range 4-117 months). BMT using FLU/low-dose CY/ATG -based regimens regardless to the donor is a beneficial therapeutic approach for FA patients.
International journal of hematology 04/2012; 95(6):675-9. · 1.17 Impact Factor
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ABSTRACT: Acute myeloid leukemia with abnormal bone marrow eosinophilia (AML-M4Eo) is often reported in core binding factor (CBF) leukemia, with translocations such as inv(16)(p13q22), t(16;16)(p13;q22) or t(8;21)(q22;q22); however, it is rarely reported with t(16;21)(q24;q22), which produces the RUNX1-CBFA2T3 (AML1-MTG16) chimera. The similarity between this chimera and RUNX1-RUNXT1 (AML1-MTG8) by t(8;21)(q22;q22) remains controversial. Adult leukemia with t(16;21)(q24;q22) was primarily therapy related, and shows poor prognosis; however, pediatric AML with this translocation was quite rare and tended to be de novo AML. We present here a 4-year-old boy with de novo AML-M4Eo and t(16;21)(q24;q22). He received chemotherapy and survived for more than 70 months without transplantation. We speculated that pediatric AML with t(16;21)(q24;q22) showed favorable prognosis, as with t(8;21)(q22;q22).
International journal of hematology 03/2012; 95(5):577-80. · 1.17 Impact Factor
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Nobuhiro Watanabe,
Yoshiyuki Takahashi, Kimikazu Matsumoto,
Yasuo Horikoshi,
Asahito Hama,
Hideki Muramatsu,
Nao Yoshida,
Hiroshi Yagasaki,
Kazuko Kudo,
Keizo Horibe,
Koji Kato,
Seiji Kojima
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ABSTRACT: Although some studies have reported that TBI and MEL offer an effective conditioning regimen for autologous SCT in acute leukemia, little has been reported regarding outcomes of allogeneic SCT. We retrospectively evaluated outcomes for 50 pediatric patients who underwent allo-SCT conditioned with intravenous MEL (180-210 mg/m(2) ) and fractionated TBI (12-13.2 Gy) from HLA-identical related donors. Nineteen patients were in CR1, 18 were in CR2, and 13 showed advanced-stage disease (≥ CR3). Patients had received allo-SCT from HLA-identical siblings (n = 45) or phenotypically HLA-identical family donors (n = 5). Median duration of follow-up for all disease-free patients was 61 months (range, 8.8-177 months). At the time of analysis, 12 patients had died. Eleven of those died of relapse, and one died of TRM. DFS rates for all patients, patients with AML (n = 12), and patients with lymphoid malignancy (n = 38) were 61.4% and 82.1%, respectively. DFS rates for CR1, CR2, and ≥CR3 cases were 89.2%, 88.1%, and 23.1%, respectively (p < 0.05). MEL/TBI for pediatric patients with hematological malignancies was associated with lower relapse rates and no increase in toxicity, resulting in better survival.
Pediatric Transplantation 07/2011; 15(6):642-9. · 1.48 Impact Factor
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ABSTRACT: This study aimed to determine the frequency of invasive fungal infection (IFI) in pediatric patients with hematologic malignancy who received amphotericin B oral suspension and early intravenous fluconazole during the neutropenic phase as prophylaxis for IFI. Records of 743 neutropenic episodes induced by cytotoxic chemotherapy between 1997 and 2008 were retrospectively reviewed to determine risk factors for and frequency of IFI. The overall frequency of IFI was 0.8% (n=6) and frequencies of proven, probable, and possible infections were 0.3% (n=2), 0.4% (n=3), and 0.1% (n=1), respectively. During 351 episodes of profound neutropenia (neutrophil count <500/μL for ≥ 14 d), overall incidence of IFI was 1.71% (n=6). Pulmonary aspergillosis was the most common causative agent, and no patients showed candidemia, or hepatosplenic candidiasis. Cytotoxic chemotherapy regimens for acute myelogenous leukemia and profound neutropenia were significantly associated with IFI (P=0.004 and P=0.009, respectively). No IFI-attributable deaths or breakthrough fungal infections occurred. Our results indicate that amphotericin B oral solution and early intravenous fluconazole may be effective in reducing the incidence and mortality of IFI in pediatric patients with hematologic malignancies.
Journal of Pediatric Hematology/Oncology 04/2011; 33(4):270-5. · 1.16 Impact Factor
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ABSTRACT: Allogeneic stem cell transplantation (SCT) is the only curative therapy for patients with refractory or relapsed acute leukemia, although the prognosis remains poor. Few reports have described outcomes of SCT in pediatric patients with refractory acute leukemia. To identify prognostic factors for these patients, we retrospectively evaluated SCT outcomes for advanced acute leukemia in 82 pediatric patients from 3 transplant units in Nagoya City between 1990 and 2008. Median age at transplantation was 8 years (range, 0.5-17 years). Transplantation was performed in the first refractory relapse for 53 patients (64.6%), in the second or subsequent relapse for 16 patients (19.5%), and during primary induction failure for 13 patients (15.9%). Only 4 patients (4.9%) underwent transplantation in the untreated first relapse, and 39 patients (47.6%) received unrelated donor progenitor cells. Of the 82 patients, 61 died (77.9%), with a median survival of 7.1 months (95% confidence interval [CI], 4.2-10.0 months). Median disease-free survival (DFS) was 4.7 months (95% CI, 2.6-6.9 months). In multivariate analysis, peripheral blood blasts, cord blood transplantation, and more than 3 courses of previous salvage chemotherapy were predictive of DFS. These results support the notion that allogeneic SCT offers only a small chance of cure for most pediatric patients with refractory or relapsed acute leukemia, and suggest that reduction of the leukemia burden and earlier optimal timing of transplantation are essential for long-term survival even in patients with refractory acute leukemia.
Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 04/2011; 17(4):516-23. · 3.15 Impact Factor
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Hideki Muramatsu,
Yoshiyuki Takahashi,
Hirotoshi Sakaguchi,
Akira Shimada,
Nobuhiro Nishio,
Asahito Hama,
Sayoko Doisaki,
Hiroshi Yagasaki, Kimikazu Matsumoto,
Koji Kato,
Seiji Kojima
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ABSTRACT: Allogeneic hematopoietic stem cell transplantation (HSCT) and tyrosine kinase inhibitor have revolutionized the treatment of patients with chronic myeloid leukemia (CML). In this study, the clinical impact of HSCT and imatinib mesylate (IM) was retrospectively analyzed in 28 children with CML treated in our institutes from 1984 to 2008. Twelve patients were given oral IM. At 36 months after initiation of IM therapy, the complete cytogenetic response rate was 90.9%, and the major molecular response rate was 36.4%. Sixteen children received allogeneic HSCT without administration of IM. The stage of disease at transplantation was: first chronic phase (n = 10), second chronic phase (n = 2), accelerated phase (n = 2), and blastic crisis (n = 2). The progression rate was significantly lower in patients treated with IM than in those treated without IM (0 vs. 28.6%, p = 0.006). In summary, the survival outcomes of pediatric patients with CML were dramatically improved by treatment with IM compared to HSCT.
International journal of hematology 02/2011; 93(2):186-91. · 1.17 Impact Factor
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ABSTRACT: Veno-occlusive disease (VOD) is a regimen-related toxicity that occurs in the early phase of hematopoietic stem cell transplantation (HSCT). Therapeutic modalities for established VOD are limited, and severe VOD characterized by multiple organ failure is associated with a fatal prognosis despite intensive supportive care.
We analyzed the data of 95 consecutive allogeneic HSCT for childhood hematological malignancies, and assessed the efficacy of our VOD prophylaxis regimen based on danaparoid (n = 48), comparing with historical control regimen based on dalteparin (n = 47).
Eight patients (danaparoid cohort in one; dalteparin cohort in seven) developed VOD on day +30 (median onset, day +22; range, day +11 to day +28) after HSCT. The probability of developing VOD for the danaparoid cohort was 2% (95% CI, 0-6%) and that of the dalteparin cohort was 15% (95% CI, 5-26%). In the Cox hazard proportional model, the danaparoid cohort had a significant advantage over the dalteparin cohort for the prophylaxis of VOD (hazard ratio (HR), 0.0; 95% CI, 0.0-0.3; P < 0.01) without increasing hemorrhagic events.
Our findings suggest that danaparoid may have promise for the prophylaxis of VOD after allogeneic HSCT and further randomized studies are warranted.
Pediatric Blood & Cancer 12/2010; 55(6):1118-25. · 1.89 Impact Factor
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Hirotoshi Sakaguchi MD,
PhD Nobuhiro Watanabe MD,
PhD Hideki Muramatsu MD,
Sayoko Doisaki MD,
PhD Nao Yoshida MD,
PhD Kimikazu Matsumoto MD,
PhD Koji Kato MD,
Hirotoshi Sakaguchi,
Nobuhiro Watanabe,
Hideki Muramatsu,
Sayoko Doisaki,
Nao Yoshida, Kimikazu Matsumoto,
Koji Kato
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ABSTRACT: Background
Veno-occlusive disease (VOD) is a regimen-related toxicity that occurs in the early phase of hematopoietic stem cell transplantation (HSCT). Therapeutic modalities for established VOD are limited, and severe VOD characterized by multiple organ failure is associated with a fatal prognosis despite intensive supportive care.ProcedureWe analyzed the data of 95 consecutive allogeneic HSCT for childhood hematological malignancies, and assessed the efficacy of our VOD prophylaxis regimen based on danaparoid (n = 48), comparing with historical control regimen based on dalteparin (n = 47).ResultsEight patients (danaparoid cohort in one; dalteparin cohort in seven) developed VOD on day +30 (median onset, day +22; range, day +11 to day +28) after HSCT. The probability of developing VOD for the danaparoid cohort was 2% (95% CI, 0–6%) and that of the dalteparin cohort was 15% (95% CI, 5–26%). In the Cox hazard proportional model, the danaparoid cohort had a significant advantage over the dalteparin cohort for the prophylaxis of VOD (hazard ratio (HR), 0.0; 95% CI, 0.0–0.3; P < 0.01) without increasing hemorrhagic events.Conclusions
Our findings suggest that danaparoid may have promise for the prophylaxis of VOD after allogeneic HSCT and further randomized studies are warranted. Pediatr Blood Cancer. 2010;55:1118–1125. © 2010 Wiley-Liss, Inc.
Pediatric Blood & Cancer 11/2010; 55(6):1118 - 1125. · 1.89 Impact Factor
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ABSTRACT: Most previous studies of graft-versus-host disease (GVHD) prophylaxis with methotrexate (MTX) alone in patients undergoing HLA-identical sibling donor bone marrow transplantation were performed in adults. With this background, we attempted to analyze the incidence and risk factors of GVHD in bone marrow transplantation (BMT) from an HLA-identical sibling donor in children with hematological malignancies using MTX alone as a prophylaxis for GVHD. Ninety-four patients received MTX by intravenous bolus injection, with a dose of 15 mg/m(2) on day +1, followed by 10 mg/m(2) on days +3, +6, and +11, and then weekly until day +60. The probability of developing grade II-IV acute GVHD and chronic GVHD was 19.1 and 31.8%, respectively. Age at transplantation and a female donor to male recipient were identified as risk factors for chronic GVHD in multivariate analysis, but no factors were identified for acute GVHD. The cumulative incidence of transplant-related mortality during the first 100 days was 9.6%. Disease-free survival at 5 years for standard- and high-risk patients was 82.1 and 39.5%, respectively. These results suggest that GVHD prophylaxis with MTX alone is safe and effective in young children under 10 years old at transplantation and in a setting other than female donor to male recipient.
International journal of hematology 01/2009; 88(5):575-82. · 1.17 Impact Factor
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Nao Yoshida,
Hiroshi Yagasaki,
Yinyan Xu,
Kazuyuki Matsuda,
Ayami Yoshimi,
Yoshiyuki Takahashi,
Asahito Hama,
Nobuhiro Nishio,
Hideki Muramatsu,
Nobuhiro Watanabe, [......],
Junichi Ueyama,
Hiroko Inada,
Hiroaki Goto,
Miharu Yabe,
Kazuko Kudo,
Junichi Mimaya,
Akira Kikuchi,
Atsushi Manabe,
Kenichi Koike,
Seiji Kojima
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ABSTRACT: Mutations in RAS, neurofibromatosis type 1 (NF1), and PTPN11, constituents of the granulocyte-macrophage colony-stimulating factor signaling pathway, have been recognized in patients with juvenile myelomonocytic leukemia (JMML). We assessed 71 children with JMML for NRAS, KRAS, and PTPN11 mutations and evaluated their clinical significance. Of the 71 patients, three had been clinically diagnosed with neurofibromatosis type 1, and PTPN11 and NRAS/KRAS mutations were found in 32 (45%) and 13 (18%) patients, respectively. No simultaneous aberrations were found. Compared with patients with RAS mutation or without any aberrations, patients with PTPN11 mutation were significantly older at diagnosis and had higher fetal Hb levels, both of which have been recognized as poor prognostic factors. As was expected, overall survival was lower for patients with the PTPN11 mutation than for those without (25 versus 64%; p = 0.0029). In an analysis of 48 patients who received hematopoietic stem cell transplantation, PTPN11 mutations were also associated with poor prognosis for survival. Mutation in PTPN11 was the only unfavorable factor for relapse after hematopoietic stem cell transplantation (p = 0.001). All patients who died after relapse had PTPN11 mutation. These results suggest that JMML with PTPN11 mutation might be a distinct subgroup with specific clinical characteristics and poor outcome.
Pediatric Research 12/2008; 65(3):334-40. · 2.70 Impact Factor
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Ayano Fujita,
Masaru Ihira,
Ryota Suzuki,
Yoshihiko Enomoto,
Hiroko Sugiyama,
Ken Sugata,
Sadao Suga,
Yoshizo Asano,
Hiroshi Yagasaki,
Seiji Kojima, Kimikazu Matsumoto,
Koji Kato,
Tetsushi Yoshikawa
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ABSTRACT: Although it has been demonstrated that human herpesvirus 6 (HHV-6) reactivation generally occurs approximately 2-3 weeks after transplantation in the hematopoietic stem cell transplantation (HSCT) recipients, the mechanism of viral reactivation remains unclear. To explore the relationship between HHV-6 reactivation and plasma cytokine levels, 24 HSCT recipients underwent measurements of plasma proinflammatory cytokine levels (IL-6, TNF-alpha, IL-1 beta, and IFN-gamma), viral isolation, and serological assays. Of these patients, 14 developed an HHV-6 reactivation, and 9 developed HHV-6 viremia approximately 2-3 weeks after transplantation. IL-6 levels were significantly higher in the recipients with an HHV-6 reactivation than in the subjects without an HHV-6 reactivation at 1 week, 2 weeks, and 4 weeks after transplantation. In addition, the level of TNF-alpha was significantly higher in recipients with an HHV-6 reactivation than in those without an HHV-6 reactivation at 2 weeks post-transplantation. Low levels of IL-1 beta and IFN-gamma were detected in a small number of the plasma samples, although there were no significant differences between the two groups in the levels of these cytokines. These results imply that proinflammatory cytokines, in particular IL-6 and TNF-alpha, play a role in the pathogenesis of HHV-6 reactivation after HSCT.
The Journal of infection 10/2008; 57(3):241-8. · 4.13 Impact Factor
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ABSTRACT: Transient leukaemia (TL) in neonates with Down syndrome (DS) is characterized by the transient appearance of blast cells in the peripheral blood that resolves spontaneously. Some TL patients die at an early age due to organ failure. Seventy DS patients with TL were studied retrospectively to identify clinical and laboratory characteristics associated with early death (<6 months of age). Sixteen of 70 patients (22.9%) died early. The main causes of death were organ failure, particularly hepatic and cardiopulmonary failure. On univariate analysis, early gestational age (EGA), high white blood cell (WBC) count (> or =100 x 10(9)/l), percentage of peripheral blasts, elevated aspartate transaminase (AST), elevated direct bilirubin (DB), and low Apgar score were significantly associated with poor survival. On multivariate analysis, EGA, WBC count, and DB were independent predictors of poor outcome. A simple risk stratification system combining EGA and WBC count was devised to predict poor outcome. Term infants (EGA > or = 37 weeks) whose WBC count was lower than 100 x 10(9)/l had the best outcome [7.7% (3/39) died early], while preterm infants (EGA < 37 weeks) whose WBC count was higher than 100 x 10(9)/l had the worst outcome [54.5% (6/11) died early]. This stratification system may be useful for identifying high-risk patients who need early therapeutic interventions.
British Journal of Haematology 06/2008; 142(4):610-5. · 4.94 Impact Factor
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Asahito Hama,
Hiroshi Yagasaki,
Yoshiyuki Takahashi,
Nobuhiro Nishio,
Hideki Muramatsu,
Nao Yoshida,
Makito Tanaka,
Hirokazu Hidaka,
Nobuhiro Watanabe,
Ayami Yoshimi, Kimikazu Matsumoto,
Kazuko Kudo,
Koji Kato,
Keizo Horibe,
Seiji Kojima
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ABSTRACT: To characterize childhood acute megakaryoblastic leukaemia (AMKL), we reviewed 45 children with AMKL diagnosed between 1986 and 2005 at Nagoya University Hospital and Japanese Red Cross Nagoya First Hospital. Twenty-four patients (53%) had AMKL associated with Down syndrome (DS-AMKL) and 21 (47%) had non-DS-AMKL. The median age of the DS-AMKL patients was 21 months (range, 8-38 months) and that of non-DS-AMKL patients was 15 months (range, 2-185 months). The morphology of blast cells was categorized into three groups according to the stage of megakaryocyte maturation. The blast cells were more immature in DS-AMKL than in non-DS-AMKL in terms of morphology and immunophenotyping. Cytogenetic abnormalities of leukaemic cells were classified into seven categories: normal karyotype including constitutional trisomy 21 in DS-AMKL; numerical abnormalities only; t(1;22)(p13;q13); 3q21q26 abnormalities; t(16;21)(p11;q22); -5/del(5q) and/or -7/del(7q); and other structural changes. The outcome of children with either DS-AMKL or non-DS-AMKL is excellent. The 10-year overall survival estimate was 79% [95% confidence interval (CI): 54-90] for DS-AMKL and 76% (95% CI: 58-91) for non-DS-AMKL (P = 0.81) with a median follow-up of 78 months (range, 20-243 months). Our study shows the diverse heterogeneity of childhood AMKL and the need for subclassification according to cytogenetic and morphological features.
British Journal of Haematology 04/2008; 140(5):552-61. · 4.94 Impact Factor
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ABSTRACT: A newborn presented with thrombocytopenia at birth and subsequently developed leukocytosis, monocytosis, and mild hepatomegaly. The bone marrow was normocellular with dysplasia and spontaneous granulocyte-monocyte colony formation was demonstrated. These findings fulfilled the diagnostic criteria of juvenile myelomonocytic leukemia. Then he developed atopic dermatitislike eczema, which led to the consideration of Wiskott-Aldrich syndrome (WAS). Lack of intracellular WASP expression and WASP gene mutation confirmed the diagnosis of WAS. After stem cell transplantation, he is alive in good condition with normal WASP expression. WAS should be considered as a differential diagnosis in male infants with juvenile myelomonocytic leukemialike features.
Journal of Pediatric Hematology/Oncology 01/2008; 29(12):836-8. · 1.16 Impact Factor
