X-linked severe combined immunodeficiency (X-SCID) with high blood levels of immunoglobulins and Aspergillus pneumonia successfully treated with micafangin followed by unrelated cord blood stem cell transplantation

Shinichi Kobayashi, Shizuko Murayama, Osamu Tatsuzawa, Goro Koinuma, Kazuteru Kawasaki, Chikako Kiyotani, Masaaki Kumagai

Research output: Contribution to journalArticle

12 Citations (Scopus)

Abstract

In this report, we describe a patient with X-linked severe combined immunodeficiency (X-SCID) who had high serum IgG, IgA, and IgM levels. The boy did well until 6 months of age, when he developed interstitial pneumonia caused by Aspergillus species, with a white cell count of 12,840/μL and only 10% lymphocytes; IgG, 991 mg/dL; IgA, 65 mg/dL; IgM, 472 mg/dL. Cell markers showed only 6.3% CD3, 2.1% CD4, 0.7% CD8, but 92% CD19 and 0.1% CD16+CD56+ (NK cells). A mutation was detected within exon 2 (C196 A→C), leading to the substitution of proline for glutamine, which has not been reported previously. The boy was successfully treated with the new antifungal drug, micafangin (MCFG), at 5 mg/kg/day for 89 days. After resolution of the pneumonia, the patient underwent successful hematopoietic stem cell transplantation with completely matched unrelated female cord blood. The CD34 stem cell dose was 3.4×104 cells/kg. In conclusion, MCFG can be a first line agent for Aspergillus pneumonia in immunocompromised hosts.

Original languageEnglish
Pages (from-to)207-210
Number of pages4
JournalEuropean Journal of Pediatrics
Volume166
Issue number3
DOIs
Publication statusPublished - 2007 Mar
Externally publishedYes

Fingerprint

Cord Blood Stem Cell Transplantation
X-Linked Combined Immunodeficiency Diseases
Aspergillus
Immunoglobulin A
Immunoglobulin M
Immunoglobulins
Pneumonia
Immunoglobulin G
Hematopoietic Stem Cell Transplantation
Interstitial Lung Diseases
Immunocompromised Host
Glutamine
Fetal Blood
Proline
Natural Killer Cells
Exons
Stem Cells
Cell Count
Lymphocytes
Mutation

Keywords

  • Cord blood transplantation
  • Micafangin
  • Paraprotein
  • X-SCID

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Cite this

X-linked severe combined immunodeficiency (X-SCID) with high blood levels of immunoglobulins and Aspergillus pneumonia successfully treated with micafangin followed by unrelated cord blood stem cell transplantation. / Kobayashi, Shinichi; Murayama, Shizuko; Tatsuzawa, Osamu; Koinuma, Goro; Kawasaki, Kazuteru; Kiyotani, Chikako; Kumagai, Masaaki.

In: European Journal of Pediatrics, Vol. 166, No. 3, 03.2007, p. 207-210.

Research output: Contribution to journalArticle

Kobayashi, Shinichi ; Murayama, Shizuko ; Tatsuzawa, Osamu ; Koinuma, Goro ; Kawasaki, Kazuteru ; Kiyotani, Chikako ; Kumagai, Masaaki. / X-linked severe combined immunodeficiency (X-SCID) with high blood levels of immunoglobulins and Aspergillus pneumonia successfully treated with micafangin followed by unrelated cord blood stem cell transplantation. In: European Journal of Pediatrics. 2007 ; Vol. 166, No. 3. pp. 207-210.
@article{72a478cb175244c8a14b34f8736a4b3b,
title = "X-linked severe combined immunodeficiency (X-SCID) with high blood levels of immunoglobulins and Aspergillus pneumonia successfully treated with micafangin followed by unrelated cord blood stem cell transplantation",
abstract = "In this report, we describe a patient with X-linked severe combined immunodeficiency (X-SCID) who had high serum IgG, IgA, and IgM levels. The boy did well until 6 months of age, when he developed interstitial pneumonia caused by Aspergillus species, with a white cell count of 12,840/μL and only 10{\%} lymphocytes; IgG, 991 mg/dL; IgA, 65 mg/dL; IgM, 472 mg/dL. Cell markers showed only 6.3{\%} CD3, 2.1{\%} CD4, 0.7{\%} CD8, but 92{\%} CD19 and 0.1{\%} CD16+CD56+ (NK cells). A mutation was detected within exon 2 (C196 A→C), leading to the substitution of proline for glutamine, which has not been reported previously. The boy was successfully treated with the new antifungal drug, micafangin (MCFG), at 5 mg/kg/day for 89 days. After resolution of the pneumonia, the patient underwent successful hematopoietic stem cell transplantation with completely matched unrelated female cord blood. The CD34 stem cell dose was 3.4×104 cells/kg. In conclusion, MCFG can be a first line agent for Aspergillus pneumonia in immunocompromised hosts.",
keywords = "Cord blood transplantation, Micafangin, Paraprotein, X-SCID",
author = "Shinichi Kobayashi and Shizuko Murayama and Osamu Tatsuzawa and Goro Koinuma and Kazuteru Kawasaki and Chikako Kiyotani and Masaaki Kumagai",
year = "2007",
month = "3",
doi = "10.1007/s00431-006-0224-y",
language = "English",
volume = "166",
pages = "207--210",
journal = "European Journal of Pediatrics",
issn = "0340-6199",
publisher = "Springer Verlag",
number = "3",

}

TY - JOUR

T1 - X-linked severe combined immunodeficiency (X-SCID) with high blood levels of immunoglobulins and Aspergillus pneumonia successfully treated with micafangin followed by unrelated cord blood stem cell transplantation

AU - Kobayashi, Shinichi

AU - Murayama, Shizuko

AU - Tatsuzawa, Osamu

AU - Koinuma, Goro

AU - Kawasaki, Kazuteru

AU - Kiyotani, Chikako

AU - Kumagai, Masaaki

PY - 2007/3

Y1 - 2007/3

N2 - In this report, we describe a patient with X-linked severe combined immunodeficiency (X-SCID) who had high serum IgG, IgA, and IgM levels. The boy did well until 6 months of age, when he developed interstitial pneumonia caused by Aspergillus species, with a white cell count of 12,840/μL and only 10% lymphocytes; IgG, 991 mg/dL; IgA, 65 mg/dL; IgM, 472 mg/dL. Cell markers showed only 6.3% CD3, 2.1% CD4, 0.7% CD8, but 92% CD19 and 0.1% CD16+CD56+ (NK cells). A mutation was detected within exon 2 (C196 A→C), leading to the substitution of proline for glutamine, which has not been reported previously. The boy was successfully treated with the new antifungal drug, micafangin (MCFG), at 5 mg/kg/day for 89 days. After resolution of the pneumonia, the patient underwent successful hematopoietic stem cell transplantation with completely matched unrelated female cord blood. The CD34 stem cell dose was 3.4×104 cells/kg. In conclusion, MCFG can be a first line agent for Aspergillus pneumonia in immunocompromised hosts.

AB - In this report, we describe a patient with X-linked severe combined immunodeficiency (X-SCID) who had high serum IgG, IgA, and IgM levels. The boy did well until 6 months of age, when he developed interstitial pneumonia caused by Aspergillus species, with a white cell count of 12,840/μL and only 10% lymphocytes; IgG, 991 mg/dL; IgA, 65 mg/dL; IgM, 472 mg/dL. Cell markers showed only 6.3% CD3, 2.1% CD4, 0.7% CD8, but 92% CD19 and 0.1% CD16+CD56+ (NK cells). A mutation was detected within exon 2 (C196 A→C), leading to the substitution of proline for glutamine, which has not been reported previously. The boy was successfully treated with the new antifungal drug, micafangin (MCFG), at 5 mg/kg/day for 89 days. After resolution of the pneumonia, the patient underwent successful hematopoietic stem cell transplantation with completely matched unrelated female cord blood. The CD34 stem cell dose was 3.4×104 cells/kg. In conclusion, MCFG can be a first line agent for Aspergillus pneumonia in immunocompromised hosts.

KW - Cord blood transplantation

KW - Micafangin

KW - Paraprotein

KW - X-SCID

UR - http://www.scopus.com/inward/record.url?scp=33847659610&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=33847659610&partnerID=8YFLogxK

U2 - 10.1007/s00431-006-0224-y

DO - 10.1007/s00431-006-0224-y

M3 - Article

C2 - 16915374

AN - SCOPUS:33847659610

VL - 166

SP - 207

EP - 210

JO - European Journal of Pediatrics

JF - European Journal of Pediatrics

SN - 0340-6199

IS - 3

ER -