Skip to content
left end
left end
right end

Conditions treated, areas of expertise & treatments offered

The Bone Marrow Transplant service at Bristol Royal Hospital for Children receives referrals from all around the UK, including Northern Ireland, Wales, Southampton, Oxford, Cambridge and all of South West England.

Areas of excellence

Transplantation of acute lymphoblastic and myeloid leukaemias, myelodysplasia, solid tumours requiring autografting, bone marrow failure syndromes (including Fanconi anaemia), adrenoleukodystrophy, metabolic diseases, osteopetrosis and haemophagocytic syndromes (HLH).

There is a particular expertise in the use of alternative donors including matched and mismatched unrelated donors, haploidentical (half-matched) parents and relatives, and cord blood donors. Detailed surveillance of patients during transplantation includes highly sensitive molecular testing for post-transplant viral infection and detailed, lineage-specific assessment of donor engraftment ("chimerism").

Further information

Specialist clinics are held both locally and throughout the South-west region. There is a specific long-term follow-up clinic for those who are further out from transplantation. Specific disease clinics also provide advice and general haematological management for acute and chronic leukaemias, bone marrow failure syndromes, adrenoleukodystrophy, Hurler syndrome and metabolic diseases.

There is close liaison with expert geneticists and laboratories providing genetic diagnosis and counselling in many of these areas.

Conditions treated

 

  • Acute Myeloid Leukaemia (high risk AML in first complete remission, in second complete remission after relapse, or with refractory disease)
  • Acute lymphoblastic leukaemia: high risk disease in first complete remission, relapsed disease in second/third complete remission
  • Chronic myeloid leukaemia: chronic phase with no good response to tyrosine kinase inhibitors or advanced phase or blast crisis
  • T-cell Non-Hodgkin Lymphoma(T-NHL)
  • Hodgkin's disease in second complete remission
  • Myelodysplasia(including juvenile myelomonocytic leukaemia [JMML], refractory anaemia, RARS, CMML, RAEBT, GATA2 deficiency*)
  • Aplastic anaemia and inherited bone marrow failure syndromes(including Fanconi anaemia, Shwachman-Diamond syndrome, dyskeratosis congenita and Diamond-Blackfan anaemia)*
  • Metabolic diseases(including early-stage adrenoleukodystrophy, fucosidosis, late-onset globoid cell leukodystrophy, a-mannosidosis, juvenile and adult onset metachromatic leukodystrophy, type I and VI mucopolysaccharidoses (Hurler's disease and Maroteaux-Lamy syndrome)
  • Osteopetrosis
  • Haemophagocytic syndromes(including HLH, Griscelli disease and Chediak-Higashi syndrome)
  • Multisystem Langerhans cell histiocytosis(LCH)
  • Thalassaemia major
  • Autograftsfor Neuroblastoma (stage IV disease and stage III disease with n-myc amplification), Atypical Teratoid/Rhabdoid Tumour (ATRT), Primitive neuroectodermal tumour (PNET), medulloblastoma, Ewing's sarcoma, germ cell tumours, rhabdomyosarcoma or Wilms tumour in appropriate high risk, relapsed or refractory disease

Diagnostic Partners:

Next Generation Sequencing (NGS) panel testing is available for the genes that cause osteopetrosis and for causes of anaemia, neutropenia (low blood neutrophil count), thrombocytopenia (low blood platelet count), pancytopenia (all blood counts reduced), bone marrow failure and genetic causes of myelodysplasia (MDS). Information on these can be accessed from Bristol Genetics Laboratory at the following links:

Osteopetrosis:

https://www.genetests.org/tests/details.php?id=149279

Treatments offered

The service has an international reputation for its pioneering work in transplantation for those lacking matched family donors, with more than 25 years of experience in this area. Services provided include assessment for suitability for transplantation, detailed patient and donor work-up investigations, transplantation in HEPA-filtered sterile rooms, immediate post-transplant aftercare (with cellular immunotherapy where required) and detailed long-term follow-up.