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Published on: May 7, 2012
Successful Cord Blood Transplantation for Idiopathic CD4+ Lymphocytopenia
Keita Yamamoto1, Yuho Najima1, Hiroko Iizuka2
1Hematology Division, Tokyo Metropolitan Cancer and Infectious Diseases Center, Komagome Hospital, Tokyo, Japan.
Insights
Idiopathic CD4+ lymphocytopenia (ICL) is a rare condition causing severe immune deficiency. A cord blood transplant (CBT) with a reduced-intensity conditioning regimen offers a potential cure for ICL.
Area of Science:
- Immunology
- Hematology
- Transplantation
Background:
- Idiopathic CD4+ lymphocytopenia (ICL) is characterized by a severe depletion of CD4+ T-lymphocytes.
- ICL predisposes patients to opportunistic infections and has an unclear etiology.
- Current therapeutic strategies for ICL lack consensus and often yield limited success.
Observation:
- A 45-year-old female patient diagnosed with ICL presented with a Mycobacterium avium complex abscess.
- Genetic evaluation using next-generation sequencing did not reveal specific gene alterations.
- The patient underwent a cord blood transplant (CBT) following a reduced-intensity conditioning (RIC) regimen.
Findings:
- Neutrophil engraftment was achieved on day +14 post-CBT.
- CD4+ lymphocyte counts normalized (>300 cells/mm3) by day +436.
- The patient remained disease-free with no sequelae at 75 months post-transplant.
Implications:
- This case demonstrates the potential of CBT with RIC as a curative treatment for ICL.
- Successful engraftment and immune reconstitution suggest CBT as a viable therapeutic option.
- Further research into ICL pathogenesis and treatment is warranted, with CBT showing promise.
Abstract:
Idiopathic CD4+ lymphocytopenia (ICL) is the depletion of CD4+ lymphocytes to <300 cells/mm3 without human immunodeficiency virus infection or other causes of lymphocytopenia. ICL causes fatal infections; its etiology remains unclear and it lacks consensus regarding therapeutic options. We report the first patient with ICL who had a successful clinical course following a cord blood transplant (CBT). A 45-year-old woman was diagnosed with ICL and underwent partial hepatectomy for an abscess caused by the Mycobacterium avium complex. No specific gene alterations were detected through next generation sequencing-based evaluation. Following a reduced-intensity conditioning (RIC) regimen consisting of fludarabine, busulfan, and 4 Gy total body irradiation, a single-unit CBT was performed. Neutrophils were engrafted on day +14. CD4+ lymphocyte counts increased to over 300 cells/mm3 on day +436. After 75 months, she was alive without any sequelae. CBT with an RIC regimen could be a curable treatment option for ICL.
