Intervention Review

You have free access to this content

Extracorporeal photopheresis versus standard treatment for acute graft-versus-host disease after haematopoietic stem cell transplantation in paediatric patients

  1. Marcus Weitz1,*,
  2. Brigitte Strahm2,
  3. Joerg J Meerpohl3,
  4. Dirk Bassler4

Editorial Group: Cochrane Childhood Cancer Group

Published Online: 25 FEB 2014

Assessed as up-to-date: 11 MAR 2013

DOI: 10.1002/14651858.CD009759.pub2


How to Cite

Weitz M, Strahm B, Meerpohl JJ, Bassler D. Extracorporeal photopheresis versus standard treatment for acute graft-versus-host disease after haematopoietic stem cell transplantation in paediatric patients. Cochrane Database of Systematic Reviews 2014, Issue 2. Art. No.: CD009759. DOI: 10.1002/14651858.CD009759.pub2.

Author Information

  1. 1

    University Children's Hospital, Pediatric Nephrology, Zurich, Switzerland

  2. 2

    University Medical School Freiburg, Pediatric Hematology and Oncology Centre for Pediatrics and Adolescent Medicine, Freiburg, Germany

  3. 3

    Medical Center - University of Freiburg, German Cochrane Centre, Freiburg, Germany

  4. 4

    University Children's Hospital, Department of Neonatology, Tuebingen, Germany

*Marcus Weitz, Pediatric Nephrology, University Children's Hospital, Steinwiesstrasse 75, Zurich, 8032, Switzerland. marcus.weitz@kispi.uzh.ch.

Publication History

  1. Publication Status: New
  2. Published Online: 25 FEB 2014

SEARCH

 
Characteristics of excluded studies [ordered by study ID]

StudyReason for exclusion

Apisarnthanarax 2003Retrospective study on 2 paediatric patients and 62 adults with cGvHD

Balda 1996Case report on 1 child with cGvHD

Besnier 1997Case series of 3 paediatric patients and 4 adults with aGvHD (2 people) and cGvHD (5 people)

Biagi 2000Case report of 2 paediatric patients with cGvHD

Bisaccia 2011Case report of 1 paediatric patient with chronic cutaneous GvHD

Child 1999Case series of 11 adults with cGvHD

D'Incan 2000Case series of 3 paediatric patients with GvHD

Dall'Amico 1997Case series of 4 paediatric patients with GvHD

Flowers 2008Multicentre prospective phase 2 randomised controlled study in people with chronic cutaneous GvHD including less than 50% paediatric patients

Foss 2003Review article reporting case series of paediatric patients with aGvHD or cGvHD treated with ECP

Foss 2005Study not randomised, not controlled. Prospective single-arm study on 23 adults and 2 paediatric patients with chronic cutaneous and visceral GvHD

Halle 2002Case series of 8 paediatric patients with cGvHD

Kanold 2003Case series of paediatric patients with cGvHD

Kanold 2005Review article reporting case series of paediatric patients with aGvHD or cGvHD treated with ECP

Looks 1997Case report of 1 paediatric patient with aGvHD

Messina 2003Prospective, not randomised, uncontrolled study of 77 paediatric patients with acute (33 children) or chronic (44 children) immunosuppressive-resistant GvHD

Perotti 1999Prospective, not randomised, uncontrolled study in 1 child with aGvHD and 6 children with cGvHD

Peters 2000Review article reporting different therapeutic options for GvHD

Rossetti 1995Case report of 1 paediatric patient with cGvHD

Rossetti 1996Case series of 9 paediatric patients with aGvHD and 8 children with cGvHD

Salvaneschi 2001Case series of paediatric patients with aGvHD and 13 children with cGvHD

Zecca 2000Review article reporting different therapeutic options for GvHD

 
Characteristics of ongoing studies [ordered by study ID]
NCT 00609609

Trial name or titleA Randomized Phase II Study for the Evaluation of Extracorporeal Photopheresis (ECP) in Combination with Corticosteroids for the Initial Treatment of Acute Graft-Versus-Host Disease (GvHD)

MethodsPhase 2, randomised, unblinded, controlled trial with a parallel design, open label

ParticipantsParticipants of both gender, no healthy volunteers accepted

Inclusion criteria

  • Participants must be recipients of allogeneic bone marrow or stem cell grafts


  • Participant must weigh above 40 kg


  • Participants must have new-onset, clinical grade II-III acute or late-acute GvHD of the GI tract or liver or the skin that developed post transplantation. The diagnosis of GvHD must be pathologically confirmed in at least 1 organ or highly suspected clinically. Pathological confirmation may occur after registration and after the start of therapy. Definition of late acute GvHD vs. acute GvHD: the diagnosis of late acute GvHD includes clinical features that are identical to acute GvHD; however, late acute GvHD is diagnosed on or after day 100 post transplantation
  • These manifestations include a maculopapular rash, abnormal liver studies (cholestatic jaundice) or nausea/vomiting/diarrhoea or a combination of these. Participants must not have any concurrent classical features of chronic GvHD in addition to the above manifestations. Features of chronic GvHD include dry eyes and mouth; contractures; sclerodermal, lichenoid skin changes; or a combination of these
  • In the clinical judgement of the principle investigator, participants must be able to sustain a platelet count and haematocrit ≥ 20,000/mL and ≥ 27%, respectively, with or without transfusions
  • Absolute white blood count>1500/mL
  • Participant must be willing to comply with all study procedures
  • All participants with childbearing potential, including males and females, must commit to using adequate contraceptive precautions throughout their participation in the study and for 3 months following the last ECP treatment


Exclusion criteria

  • Participants developing chronic GvHD following immunomodulation with immunosuppression withdrawal or donor lymphocyte infusion
  • Any clinical manifestation consistent with de novo chronic GvHD or overlapped syndrome of acute and chronic GvHD
  • Participants who are unable to tolerate the volume shifts associated with ECP treatment due to the presence of any of the following conditions: uncompensated congestive heart failure, pulmonary oedema, severe asthma or chronic obstructive pulmonary disease, hepatorenal syndrome
  • Active bleeding
  • International normalised ratio > 2
  • Participants cannot have received methylprednisolone > 2 mg/kg/day for more than 72 hours prior to registration
  • Participants cannot have received any other immunosuppression for treatment of GvHD other than calcineurin inhibitors and corticosteroids. Participants are allowed to have had any GvHD prophylaxis with the exception of ECP
  • Participants with known hypersensitivity or allergy to psoralen
  • Participants with known hypersensitivity or allergy to both citrate and heparin
  • Participants with co-existing photosensitive disease (e.g. porphyria, systemic lupus erythematosus, albinism) or coagulation disorders
  • Uncontrolled, persistent hypertriglyceridaemia (levels > 800 mg/dl)

InterventionsArm 1: corticosteroids; drug: methylprednisolone 2 mg/kg/day with a taper to no less than 1 mg/kg/day by day 14, followed by a tapering schedule according to the suggested guidelines (trade names: Medrol, Depo-Medrol, Solu-Medrol)

Arm 2: ECP plus corticosteroids; procedure: photopheresis 8-9 photopheresis treatments weekly for days 1-14, 6 treatments weekly from days 15-28, and then 2 treatments weekly until day 60. After day 60, the doctor will decide whether ECP is worth continuing, and the frequency of treatments; drug: methylprednisolone 2 mg/kg/day with a taper to no less than 1 mg/kg/day by day 14, followed by a tapering schedule according to the suggested guidelines (trade names: Medrol, Depo-Medrol, Solu-Medrol)

OutcomesTreatment failure (time frame: first analysis after first 20 participants; acute GvHD will be scored every week for 8 weeks

Starting dateJanuary 2008 to January 2015

Contact informationAmin Alousi, MD from M.D. Anderson Cancer Center, Houston, TX, US

NotesParticipants (< 18 years of age) are eligible for this study if their body weight is > 40 kg. It is uncertain how many children will be included in this study. In September 2012, the number of the participants < 18 years of age is < 50% (September 2012)