Increased risk of gastrointestinal acute GVHD following the addition of melphalan to busulfan/cyclophosphamide conditioning

Pediatr Transplant. 2013 May;17(3):285-93. doi: 10.1111/petr.12061. Epub 2013 Mar 14.


Risk factors associated with the development of aGVHD in the gastrointestinal tract have not been studied in depth. We retrospectively assessed 25 pediatric patients with MDS and JMML and compared the treatment outcome of two different conditioning regimens. Seventeen children (68%) underwent conditioning with busulfan (Bu), cyclophosphamide (Cy), and melphalan (Mel) and eight children (32%) with Bu and Cy. Gastrointestinal aGVHD stages II-IV (day 0-100) were observed in 47% (eight of 17) of the patients in the BuCyMel group and in none (0 of 8) in the BuCy group (p < 0.05). In patients who developed gastrointestinal aGVHD stages III-IV, a 24-h variation in the Bu concentration with a nighttime peak was noted. HC and liver aGVHD stages II-IV were observed in 47% (eight of 17) and 35% (six of 17) after BuCyMel conditioning and in 0% (0 of 17) and 12.5% (one of eight) after BuCy conditioning. The overall survival rate was 53% (nine of 17) in the BuCyMel group and 62.5% (five of eight) in the BuCy group. In conclusion, the addition of melphalan to the BuCy conditioning regimen resulted in severe gastrointestinal complications and did not improve overall survival.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Busulfan / administration & dosage
  • Busulfan / adverse effects*
  • Child
  • Child, Preschool
  • Cyclophosphamide / adverse effects*
  • Female
  • Gastrointestinal Tract / drug effects
  • Graft vs Host Disease / diagnosis*
  • Hematopoietic Stem Cell Transplantation / adverse effects
  • Hematopoietic Stem Cell Transplantation / methods
  • Humans
  • Infant
  • Leukemia, Myelomonocytic, Acute / therapy
  • Male
  • Melphalan / administration & dosage
  • Melphalan / adverse effects*
  • Myelodysplastic Syndromes / therapy
  • Retrospective Studies
  • Risk Factors
  • Transplantation Conditioning / adverse effects*
  • Transplantation Conditioning / methods
  • Treatment Outcome


  • Cyclophosphamide
  • Busulfan
  • Melphalan