PMID- 27107757 OWN - NLM STAT- MEDLINE DCOM- 20170313 LR - 20211204 IS - 1865-3774 (Electronic) IS - 0925-5710 (Linking) VI - 104 IP - 1 DP - 2016 Jul TI - Immunosuppressive therapy for patients with Down syndrome and idiopathic aplastic anemia. PG - 130-3 LID - 10.1007/s12185-016-1997-z [doi] AB - Idiopathic aplastic anemia (AA) is a rare hematological complication of Down syndrome (DS). The safety and efficacy of immunosuppressive therapy (IST) in individuals with DS remain unknown. We used a standard regimen of IST, comprising antithymocyte globulin and cyclosporine A, to treat three children with DS and idiopathic acquired AA. Two patients achieved a hematological (complete or partial) response and became transfusion independent at the final follow-up. The third patient failed to respond to IST and underwent bone marrow transplantation from a human leukocyte antigen (HLA)-mismatched unrelated donor. None of the patients experienced severe or unexpected adverse events during IST. Our experience suggests that IST is a safe and reasonable treatment, even in individuals with DS who suffer from AA and lack an HLA-matched sibling donor. FAU - Suzuki, Kyogo AU - Suzuki K AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan. FAU - Muramatsu, Hideki AU - Muramatsu H AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan. FAU - Okuno, Yusuke AU - Okuno Y AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan. FAU - Narita, Atsushi AU - Narita A AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan. FAU - Hama, Asahito AU - Hama A AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan. FAU - Takahashi, Yoshiyuki AU - Takahashi Y AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan. FAU - Yoshida, Makoto AU - Yoshida M AD - Department of Pediatrics, Japanese Red Cross Asahikawa Hospital, Asahikawa, Japan. FAU - Horikoshi, Yasuo AU - Horikoshi Y AD - Division of Hematology and Oncology, Shizuoka Children's Hospital, Shizuoka, Japan. FAU - Watanabe, Ken-Ichiro AU - Watanabe K AD - Division of Hematology and Oncology, Shizuoka Children's Hospital, Shizuoka, Japan. FAU - Kudo, Kazuko AU - Kudo K AD - Department of Pediatrics, Fujita Health University School of Medicine, Toyoake, Japan. FAU - Kojima, Seiji AU - Kojima S AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan. kojimas@med.nagoya-u.ac.jp. LA - eng PT - Journal Article DEP - 20160423 PL - Japan TA - Int J Hematol JT - International journal of hematology JID - 9111627 RN - 0 (Antilymphocyte Serum) RN - 0 (Immunosuppressive Agents) RN - 83HN0GTJ6D (Cyclosporine) SB - IM MH - Anemia, Aplastic/etiology/*therapy MH - Antilymphocyte Serum/therapeutic use MH - Bone Marrow Transplantation MH - Child MH - Cyclosporine/therapeutic use MH - Down Syndrome/complications/*therapy MH - Humans MH - Immunosuppression Therapy/methods MH - Immunosuppressive Agents/*therapeutic use MH - Treatment Outcome OTO - NOTNLM OT - Aplastic anemia OT - Down syndrome OT - Immunosuppressive therapy EDAT- 2016/04/25 06:00 MHDA- 2017/03/14 06:00 CRDT- 2016/04/25 06:00 PHST- 2016/02/04 00:00 [received] PHST- 2016/03/29 00:00 [accepted] PHST- 2016/03/29 00:00 [revised] PHST- 2016/04/25 06:00 [entrez] PHST- 2016/04/25 06:00 [pubmed] PHST- 2017/03/14 06:00 [medline] AID - 10.1007/s12185-016-1997-z [pii] AID - 10.1007/s12185-016-1997-z [doi] PST - ppublish SO - Int J Hematol. 2016 Jul;104(1):130-3. doi: 10.1007/s12185-016-1997-z. Epub 2016 Apr 23.