Faculty Profiles - MURAMATSU, Hideki

Papers - MURAMATSU, Hideki

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Single-Cell Transcriptomic Analysis of Epstein-Barr Virus-Associated Hemophagocytic Lymphohistiocytosis Reviewed

Takako Suzuki, Yoshitaka Sato, Yusuke Okuno, Yuka Torii, Yuto Fukuda, Kazunori Haruta, Makoto Yamaguchi, Yoshiki Kawamura, Asahito Hama, Atsushi Narita, Hideki Muramatsu, Tetsushi Yoshikawa, Yoshiyuki Takahashi, Hiroshi Kimura, Yoshinori Ito, Jun-ichi Kawada

Journal of Clinical Immunology Vol. 44 ( 4 ) page： 103 2024.4

Nanopore sequencing in distinguishing between wild-type and vaccine strains of Varicella-Zoster virus. Reviewed International journal

Yuto Fukuda, Takako Suzuki, Ken-Ichi Iwata, Kazunori Haruta, Makoto Yamaguchi, Yuka Torii, Atsushi Narita, Hideki Muramatsu, Yoshiyuki Takahashi, Jun-Ichi Kawada

Vaccine Vol. 42 ( 11 ) page： 2927 - 2932 2024.3

Language：English Publishing type：Research paper (scientific journal)

BACKGROUND: The introduction of varicella vaccines into routine pediatric immunization programs has led to a considerable reduction in varicella incidence. However, there have been reports of varicella, herpes zoster, and meningitis caused by the vaccine strain of varicella-zoster virus (VZV), raising concerns. Establishing the relationship between the wild-type and vaccine strains in VZV infections among previously vaccinated individuals is crucial. Differences in the single nucleotide polymorphisms (SNPs) among vaccine strains can be utilized to identify the strain. In this study, we employed nanopore sequencing to identify VZV strains and analyzed clinical samples. METHODS: We retrospectively examined vesicle and cerebrospinal fluid samples from patients with VZV infections. One sample each of the wild-type and vaccine strains, previously identified using allelic discrimination real-time PCR and direct sequencing, served as controls. Ten samples with undetermined VZV strains were included. After DNA extraction, a long PCR targeting the VZV ORF62 region was executed. Nanopore sequencing identified SNPs, allowing discrimination between the vaccine and wild-type strains. RESULTS: Nanopore sequencing confirmed SNPs at previously reported sites (105,705, 106,262, 107,136, and 107,252), aiding in distinguishing between wild-type and vaccine strains. Among the ten unknown samples, nine were characterized as wild strains and one as a vaccine strain. Even in samples with low VZV DNA levels, nanopore sequencing was effective in strain identification. CONCLUSION: This study validates that nanopore sequencing is a reliable method for differentiating between the wild-type and vaccine strains of VZV. Its ability to produce long-read sequences is remarkable, allowing simultaneous confirmation of known SNPs and the detection of new mutations. Nanopore sequencing can serve as a valuable tool for the swift and precise identification of wild-type and vaccine strains and has potential applications in future VZV surveillance.

DOI： 10.1016/j.vaccine.2024.03.046

PubMed

Novel mutation in <i>KIF23</i> causing congenital dyserythropoietic anemia type III in patients who underwent allogeneic hematopoietic stem cell transplantation Reviewed

Tomoko Fujikawa, Suguru Uemura, Daiichiro Hasegawa, Naoya Morisada, Sayaka Nakamura, Aiko Kozaki, Atsuro Saito, Kenji Kishimoto, Toshiaki Ishida, Takeshi Mori, Kimikazu Yakushijin, Yoshio Katayama, Daiki Yamashita, Manabu Wakamatsu, Motoharu Hamada, Hideki Muramatsu, Yoshiyuki Takahashi, Yoshiyuki Kosaka

Pediatric Blood & Cancer Vol. 71 ( 6 ) page： e30736 2024.3

Pharmacokinetics, safety, and efficacy of 20% subcutaneous immunoglobulin (Ig20Gly) administered weekly or every 2 weeks in Japanese patients with primary immunodeficiency diseases: a phase 3, open-label study Reviewed

Hirokazu Kanegane, Akifumi Endo, Satoshi Okada, Hidenori Ohnishi, Masataka Ishimura, Ryuta Nishikomori, Kohsuke Imai, Shigeaki Nonoyama, Hideki Muramatsu, Taizo Wada, Atsushi Kuga, Ko Sakamoto, Sharon Russo-Schwarzbaum, Liang-Hui Chu, Barbara McCoy, Zhaoyang Li, Leman Yel

Immunotherapy Advances 2024.3

A retrospective analysis of gene fusions and treatment outcomes in pediatric acute megakaryoblastic leukemia without Down syndrome. Reviewed International journal

Kyogo Suzuki, Asahito Hama, Yusuke Okuno, Yinyan Xu, Atsushi Narita, Nao Yoshida, Hideki Muramatsu, Nobuhiro Nishio, Koji Kato, Seiji Kojima, Keon Hee Yoo, Yoshiyuki Takahashi

Haematologica Vol. 109 ( 6 ) page： 1936 - 1940 2024.2

Myeloproliferative disorder in a patient with RIT1-associated Noonan syndrome: Case report and literature review. Reviewed

Suzuki K, Wakamatsu M, Ito Y, Ishikawa M, Shimotakahara A, Futagawa H, Yamamoto Y, Nagamine H, Saito O, Muramatsu H, Yuza Y

Pediatric blood & cancer Vol. 71 ( 2 ) page： e30780 2024.2

愛知県における副腎白質ジストロフィー新生児マススクリーニングの2年間の実績と課題 Reviewed

中田智彦, 伊藤祐史, 若松学, 村松秀城, 山本啓之, 城所博之, 久保田一生, 山田晋一郎, 勝野雅央, 伊藤哲哉, 下澤伸行, 高橋義行, 夏目淳

日本小児科学会雑誌 Vol. 128 ( 2 ) page： 283 - 283 2024.2

Allogeneic Hematopoietic Cell Transplantation for Juvenile Myelomonocytic Leukemia with a Busulfan, Fludarabine, and Melphalan Regimen: JPLSG JMML-11. Reviewed International journal Open Access

Kazuo Sakashita, Nao Yoshida, Hideki Muramatsu, Yoshitoshi Ohtsuka, Kenichiro Watanabe, Miharu Yabe, Harumi Kakuda, Yuko Honda, Tomoyuki Watanabe, Masami Haba, Shigeru Ohmori, Kazuyuki Matsuda, Yuki Yuza, Akiko Saito, Keizo Horibe, Souichi Adachi, Atsushi Manabe

Transplantation and cellular therapy Vol. 30 ( 1 ) page： 105.e1-105.e10 - 105.e10 2024.1

Language：English Publishing type：Research paper (scientific journal)

Juvenile myelomonocytic leukemia (JMML), which is classified as a myelodysplastic/myeloproliferative neoplasm, is a rare hematologic malignancy of childhood. Most patients with JMML require allogeneic hematopoietic cell transplantation (HCT) as a curative therapy. A Japanese retrospective analysis demonstrated favorable outcomes for a busulfan (BU) + fludarabine (FLU) + melphalan (MEL) regimen, with an overall survival (OS) of 72% and an event-free survival (EFS) of 53%. To further validate the efficacy and safety of this regimen, the Japan Pediatric Leukemia/Lymphoma Study Group (JPLSG) conducted a nationwide prospective study, JMML-11. Between July 2011 and June 2017, 28 patients with newly diagnosed JMML were enrolled in JMML11. Low-dose chemotherapy for tumor control before HCT was recommended, and patients treated with AML-type chemotherapy and azacitidine were excluded. The conditioning regimen comprised i.v. BU, 16 doses administered every 6 h, with dose adjustment based on pharmacokinetic (PK) studies on days -11 to -8; FLU, 30 mg/m2/day or 1 mg/kg/day for patients <10 kg or age <1 year on days -7 to -4; and MEL, 90 mg/m2/day or 3 mg/kg/day for patients <10 kg or <1 year on days -3 to -2. The donor was selected by the physician in charge. A family donor was available for 7 patients (3 HLA-matched siblings, 3 HLA-1-antigen mismatched parents, and 1 haploidentical father). Overall, 21 patients received grafts from unrelated donors, including 8 HLA-matched donors and 13 HLA-mismatched donors. The graft source was related bone marrow (BM) for 7 patients, unrelated BM for 14 patients, and unrelated cord blood for 7 patients. Neutrophil engraftment was achieved in 21 of 28 patients (75%), with a median of 20.5 days (range, 11 to 39 days) after transplantation. The 3-year OS, 3-year EFS, 3-year relapse rate, and 3-year transplantation-related mortality were 63% (95% confidence interval [CI], 42% to 78%), 52% (95% CI, 32% to 69%), 18% (95% CI, 6% to 34%), and 21% (95% CI, 9% to 38%), respectively. WBC count before the conditioning regimen (≥7.0 × 109/L) was significantly associated with inferior EFS and OS. Body surface area ≥.5 m2, spleen size <4 cm before conditioning, and HLA-matched unrelated BM donors were significantly associated with better OS. Adverse effects related to the conditioning regimen included febrile neutropenia (86%), diarrhea (39%), hypoxemia (21%), and mucositis (18%). BU-associated toxicity, including sinusoidal obstruction syndrome (SOS) and thrombotic microangiopathy (TMA), occurred in 7 patients (25%; SOS, n = 6; TMA, n = 2). Retrospective analysis of PK data after the first BU dose in 23 patients, including 6 with SOS and 17 without SOS, did not show significant differences between groups. The JMML-11 study confirms the positive results of previous retrospective analyses. BU+FLU+MEL might become a standard conditioning regimen for patients with JMML.

DOI： 10.1016/j.jtct.2023.10.002

Open Access

PubMed

Risk factors of bloodstream infection after allogeneic hematopoietic cell transplantation in children/adolescent and young adults. Reviewed Open Access

Sajiki D, Muramatsu H, Wakamatsu M, Yamashita D, Maemura R, Tsumura Y, Imaya M, Yamamori A, Narita K, Kataoka S, Taniguchi R, Narita A, Nishio N, Takahashi Y

PloS one Vol. 19 ( 8 ) page： e0308395 2024

Two cases of AMeD syndrome with isochromosome 1q treated with allogeneic stem cell transplantation. Reviewed

Kagajo M, Moritani K, Iwamoto M, Miyamoto M, Imai T, Hamada M, Wakamatsu M, Muramatsu H, Eguchi-Ishimae M, Eguchi M

Leukemia research reports Vol. 22 page： 100476 2024

Noonan Syndrome-related Myeloproliferative Disorder Occurring in the Neonatal Period: Case Report and Literature Review. Reviewed International journal

Yuki Hoshino, Kunihiko Moriya, Kanako Mitsui-Sekinaka, Yu Hashimoto, Satoshi Nakayama, Daichi Sajiki, Hideki Muramatsu, Hidetoshi Hagiwara, Shuichi Suzuki, Yujin Sekinaka, Hajime Wakamatsu, Hiroyuki Kawaguchi, Kohsuke Imai

Journal of pediatric hematology/oncology 2023.12

Language：English Publishing type：Research paper (scientific journal)

Noonan syndrome-related myeloproliferative disorder (NS/MPD) and juvenile myelomonocytic leukemia (JMML) are rare MPDs that occur in young children. We herein report a case of NS/MPD with neonatal onset. The patient had a characteristic appearance and high monocyte count in the peripheral blood and bone marrow. Genetic testing showed the E139D mutation in PTPN11; however, the patient did not meet all the diagnostic criteria for JMML, and we thus diagnosed him with NS/MPD. Eight other cases of NS/MPD with neonatal onset are also summarized. The initial presentation varied, and the prognosis was considered poor compared with previous reports of NS/MPD.

DOI： 10.1097/MPH.0000000000002803

PubMed

A Japanese retrospective study of non-tuberculous mycobacterial infection in children, adolescents, and young adult patients with hematologic-oncologic diseases. Reviewed International journal

Yusuke Tsumura, Hideki Muramatsu, Nobuyuki Tetsuka, Takahiro Imaizumi, Kikue Sato, Kento Inoue, Yoshitomo Motomura, Yuko Cho, Daiki Yamashita, Daichi Sajiki, Ryo Maemura, Ayako Yamamori, Masayuki Imaya, Manabu Wakamatsu, Kotaro Narita, Shinsuke Kataoka, Motoharu Hamada, Rieko Taniguchi, Eri Nishikawa, Atsushi Narita, Nobuhiro Nishio, Seiji Kojima, Yoshihiko Hoshino, Yoshiyuki Takahashi

Haematologica 2023.10

Language：English Publishing type：Research paper (scientific journal)

Non-tuberculous mycobacterial infection (NTM) is rare in healthy children, with lymphadenitis being the most common presentation. Immunocompromised populations are known to be at high risk, but the clinical picture of NTM infection in pediatric hematology/oncology patients is unclear. In this nationwide retrospective analysis of patients under the age of 40 treated in Japanese pediatric hematology/oncology departments who developed NTM infection between January 2010 and December 2020, 36 patients (21 patients with hematopoietic stem cell transplantation (HSCT) and 15 nontransplant patients) were identified. Post-transplant patients were infected with NTM at 24 sites, including the lungs (n = 12), skin and soft tissues (n = 6), bloodstream (n = 4), and others (n = 2). Nine of twelve patients with pulmonary NTM infection had a history of pulmonary graft-versus-host disease (GVHD), and rapid-growing mycobacteria (RGM) were isolated from five of them. In nontransplant patients, the primary diseases were acute lymphoblastic leukemia (ALL; n = 5), inborn errors of immunity (IEI; n = 6), and others (n = 4). All cases of ALL had bloodstream infections with RGM, whereas all cases of IEI were infected with slow-growing mycobacteria (SGM). In summary, three typical clinical scenarios for pediatric hematology/oncology patients have been established: RGM-induced pulmonary disease in patients with pulmonary GVHD, RGM bloodstream infection in patients with ALL, and SGM infection in patients with IEI. Our findings suggest that NTM must be regarded as a pathogen for infections in these high-risk patients, especially those with pulmonary GVHD, who may require active screening for NTM.

DOI： 10.3324/haematol.2023.283636

PubMed

Severe β-thalassemia (Hb Zunyi) mimicking congenital dyserythropoietic anemia. Reviewed International journal

Jun Nagahama, Takuro Nishikawa, Tatsuro Nakamura, Shunsuke Nakagawa, Yuichi Kodama, Hideyuki Terazono, Manabu Wakamatsu, Hideki Muramatsu, Yasuhiro Yamashiro, Hitoshi Kanno, Yasuhiro Okamoto

Pediatric blood & cancer Vol. 70 ( 12 ) page： e30706 2023.10

Successful treatment of DOCK8 deficiency by allogeneic hematopoietic cell transplantation from alternative donors Reviewed

Kono A., Wakamatsu M., Umezawa Y., Muramatsu H., Fujiwara H., Tomomasa D., Inoue K., Hattori K., Mitsui T., Takada H., Minegishi Y., Takahashi Y., Yamamoto M., Mori T., Kanegane H.

International Journal of Hematology Vol. 118 ( 4 ) page： 519 - 525 2023.10

Publisher：International Journal of Hematology

Dedicator of cytokinesis 8 (DOCK8) deficiency is a rare autosomal recessive inborn error of immunity (IEI) characterized by eczematous dermatitis, elevated serum IgE, and recurrent infections, comprising a seemingly hyper-IgE syndrome (HIES). DOCK8 deficiency is only curable with allogeneic hematopoietic cell transplantation (HCT), but the outcome of HCT from alternative donors is not fully understood. Here, we describe the cases of two Japanese patients with DOCK8 deficiency who were successfully treated by allogeneic HCT from alternative donors. Patient 1 underwent cord blood transplantation at the age of 16 years, and Patient 2 underwent haploidentical peripheral blood stem cell transplantation with post-transplant cyclophosphamide at the age of 22 years. Each patient received a fludarabine-based conditioning regimen. Their clinical manifestations, including refractory molluscum contagiosum, promptly improved post-HCT. They achieved successful engraftment and immune reconstitution without serious complications. Alternative donor sources such as cord blood and haploidentical donors can be options for allogeneic HCT for DOCK8 deficiency.

DOI： 10.1007/s12185-023-03613-y

Scopus

Pediatric erythroblastic transformation of JAK2-mutated prefibrotic primary myelofibrosis with concurrent PHF6 mutations Reviewed

Oshiro T., Hamada S., Kiyuna S., Sakiyama H., Hyakuna N., Tamaki T., Muramatsu H., Nakanishi K.

Pediatric Blood and Cancer Vol. 70 ( 10 ) 2023.10

Updated guidelines for chronic active Epstein–Barr virus disease Reviewed

Jun-ichi Kawada, Yoshinori Ito, Koichi Ohshima, Masaki Yamada, Shinsuke Kataoka, Hideki Muramatsu, Akihisa Sawada, Taizo Wada, Ken-Ichi Imadome, Ayako Arai, Keiji Iwatsuki, Shouichi Ohga, Hiroshi Kimura

International Journal of Hematology 2023.9

Publishing type：Research paper (scientific journal) Publisher：Springer Science and Business Media LLC

Abstract

Chronic active Epstein–Barr virus disease (CAEBV), formerly named chronic active Epstein–Barr virus infection, is characterized by systemic inflammation and clonal proliferation of Epstein–Barr virus (EBV)-infected T or NK cells. As CAEBV is a potentially life-threatening illness, appropriate diagnosis and therapeutic interventions are necessary for favorable clinical outcomes. Substantial evidence regarding the pathogenesis and treatment of CAEBV has been accumulated since previous guidelines for the diagnosis of CAEBV were proposed. To reflect this evidence, we updated the guidelines for the diagnosis and treatment of CAEBV to improve clinical management of the disease. The details of the updated guidelines are presented in this report. Diagnosis of CAEBV now requires confirmation of a high copy number of EBV genome and EBV-infected T or NK cells. An EBV DNA load ≥ 10,000 IU/mL in whole blood is proposed as the diagnostic cutoff value for CAEBV in this updated guideline. A standard treatment approach for CAEBV has not been established, and hematopoietic stem cell transplantation (HSCT) is considered the only curative treatment. Chemotherapy can be administered to control disease activity before HSCT.

DOI： 10.1007/s12185-023-03660-5

Scopus

Other Link： https://link.springer.com/article/10.1007/s12185-023-03660-5/fulltext.html

Vedolizumab for children with intestinal graft-versus-host disease: a case report and literature review Reviewed

Fukuta T., Muramatsu H., Yamashita D., Sajiki D., Maemura R., Tsumura Y., Yamamori A., Imaya M., Wakamatsu M., Nishikawa E., Narita K., Kataoka S., Taniguchi R., Narita A., Nishio N., Takahashi Y.

International Journal of Hematology Vol. 118 ( 3 ) page： 411 - 417 2023.9

Publisher：International Journal of Hematology

Acute graft-versus-host disease (aGVHD) is a challenging complication of allogeneic hematopoietic stem cell transplantation, and alternative therapies for patients showing inadequate response to steroids are limited. Vedolizumab, an anti-α4β7 integrin antibody widely used for treating inflammatory bowel diseases, has recently been studied in adult patients with steroid-refractory intestinal aGVHD. However, few studies have examined its safety and effectiveness in pediatric patients with intestinal aGVHD. We report the case of a male patient with intestinal late-onset aGVHD treated with vedolizumab. He underwent allogeneic cord blood transplantation for warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome and developed intestinal late-onset aGVHD 31 months after transplantation. The patient was refractory to steroids; however, vedolizumab was initiated 43 months after transplantation (at the age of 7 years) and the symptoms of intestinal aGVHD were alleviated. Additionally, favorable endoscopic findings were observed, such as reduction of erosion and regenerative epithelial growth. We also evaluated the efficacy of vedolizumab in 10 patients with intestinal aGVHD (9 from the literature review and the present case). Six patients (60%) showed an objective response to vedolizumab. No serious adverse events were observed in any patients. Vedolizumab is a potential treatment option for steroid-refractory intestinal aGVHD in pediatric patients.

DOI： 10.1007/s12185-023-03590-2

Scopus

Two Cases of Juvenile Myelomonocytic Leukemia and Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease Reviewed Open Access

Yamamoto H., Natsume J., Kaneko K., Takahashi T., Wakamatsu M., Ogawa C., Kumai S., Suzui R., Sawamura F., Shiraki A., Nakata T., Kidokoro H., Muramatsu H., Takahashi Y.

Pediatric Neurology Vol. 144 page： 1 - 4 2023.7

Publisher：Pediatric Neurology

Background: Myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOGAD) is an autoimmune demyelinating disorder that often manifests after infections or vaccinations. We report two patients who developed MOGAD out of eight patients with juvenile myelomonocytic leukemia (JMML) that has never been reported. Methods: We investigated two patients with JMML who developed MOGAD among 127 patients with leukemia from 2012 to 2021. Results: Patient 1 was treated for JMML and developed fever and impaired consciousness at two years and one month of age. Magnetic resonance imaging revealed high-intensity lesions in the left frontal and left occipital white matter. The serum anti-MOG antibody test was positive, while the test was negative in the stored serum 45 days before the onset of encephalopathy. He had relapse of MOGAD after steroid therapy and plasmapheresis. Patient 2, who was treated for JMML, became apathetic and mute at three years and seven months of age. Magnetic resonance imaging revealed left frontoparietal subcortical high-intensity lesions. Anti-MOG antibody at the onset of encephalopathy was positive, while it was negative in stored serum 57 days before and 47 days after the onset. Conclusion: We treated two patients who developed MOGAD out of eight patients with JMML and none with MOGAD out of 119 patients with acute lymphocytic leukemia, acute myelocytic leukemia, or chronic myelocytic leukemia. The activated autoimmune process via the RAS pathway abnormality may have led to the formation of the anti-MOG antibody and the onset of MOGAD. MOGAD can occur in children with JMML, and abnormalities of the RAS pathway possibly contribute to its onset.

DOI： 10.1016/j.pediatrneurol.2023.03.002

Open Access

Scopus

Human leukocyte antigen 7/8-matched unrelated bone marrow transplantation using anti-thymocyte globulin in children Reviewed

Hamada M., Muramatsu H., Torii Y., Suzuki K., Narita A., Yoshida T., Imaya M., Yamamori A., Wakamatsu M., Miwata S., Narita K., Kataoka S., Kawashima N., Taniguchi R., Nishikawa E., Nishio N., Ito Y., Kojima S., Takahashi Y.

International Journal of Hematology Vol. 118 ( 1 ) page： 125 - 130 2023.7

Publisher：International Journal of Hematology

Human leukocyte antigen (HLA) mismatched unrelated donor transplantation is associated with an increased risk of graft-versus-host disease, graft failure, and infection, which increases post-transplant morbidity and mortality. In this single-center retrospective study, outcomes were evaluated in 30 consecutive children who underwent bone marrow transplantation (BMT) from HLA 1 allele-mismatched (HLA 7/8-matched) unrelated donors with rabbit anti-thymocyte globulin (rATG) as graft-versus-host disease (GVHD) prophylaxis. The 3-year overall survival (OS), event-free survival (EFS), and GVHD-relapse-free survival rates were 91.7% (95% CI 70.5%–91.9%), 88.3% (95% CI 67.5%–96.1%), and 73.9% (95% CI 52.4%–86.8%), respectively. Grade II–IV and III–IV acute GVHD occurred in 10 (33%) and 2 (7.0%) patients, respectively. The 3-year cumulative incidence of chronic GVHD was 7.8%. No fatal viral infections occurred. The study results show the feasibility of HLA 7/8-matched unrelated BMT with ATG to achieve favorable outcomes and acceptable GVHD, especially for patients who lack a fully matched donor.

DOI： 10.1007/s12185-023-03571-5

Scopus

Successful living-donor liver transplantation for sustained liver failure even after resolution of infiltrative massive hepatic invasion of stage 4S neuroblastoma: a case report. Reviewed International journal Open Access

Kanta Jobara, Ayako Yamamori, Masato Shizuku, Nobuhiko Kurata, Yasuhiro Fujimoto, Hideki Muramatsu, Yoshiyuki Takahashi, Yasuhiro Ogura

Surgical case reports Vol. 9 ( 1 ) page： 100 - 100 2023.6

Language：English Publishing type：Research paper (scientific journal)

BACKGROUND: Neuroblastoma is the most common extracranial solid tumor in childhood. Stage 4S neuroblastoma is a unique subset of neuroblastoma characterized by a favorable course and potentially low malignancy with a high rate of spontaneous tumor regression. However, recent reports have shown that there is a subgroup of patients with stage 4S neuroblastoma characterized by MYCN amplification, chromosomal aberrations, age of < 2 months at diagnosis, and significantly poorer outcomes. CASE PRESENTATION: A 1-month-old male infant with a huge abdominal tumor was transferred to our hospital and diagnosed with stage 4S neuroblastoma. The patient showed respiratory distress due to abdominal compartment syndrome secondary to massive hepatic invasion, and he required a silo operation and mechanical ventilation. After chemotherapy using carboplatin and etoposide, the infiltrative massive hepatic invasion was resolved and the abdominal compartment syndrome gradually improved; however, liver dysfunction as evidenced by hyperbilirubinemia, coagulopathy, and hyperammonemia continued. At the age of 3 months, living-donor liver transplantation was performed for treatment of sustained liver failure using a reduced lateral segment graft from the patient's father. Post-transplant liver function recovered immediately. Examination of the explanted liver demonstrated that the majority of liver tissue had been replaced by fibroblastic cells after massive hepatocyte dropout. There were only small areas of residual neuroblastoma cells in the liver specimen. The patient was discharged from the hospital 5 months after transplantation with home intermittent respiratory support. At the time of this writing (23 months after liver transplantation), he was in good condition with no signs of recurrence of neuroblastoma. CONCLUSIONS: We have herein presented a case of successful pediatric living-donor liver transplantation for sustained liver failure even after resolution of infiltrative massive hepatic invasion of stage 4S neuroblastoma. Our case clearly shows that liver transplantation can be added as an appropriate extended treatment option for liver failure after resolution of stage 4S neuroblastoma.

DOI： 10.1186/s40792-023-01681-0

Open Access

PubMed

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Papers - MURAMATSU, Hideki