Clinical Services U
p on the sixth floor at St Mary’s Paddington the paediatric day unit is a cheerful place. Bright and airy, decorated with children’s artwork, it is a reassuring environment
for youngsters suffering from non-malignant blood disorders such as aplastic anaemia, inherited bone marrow failure syndromes, sickle cell disease and thalassemia. In charge of the unit is Dr Josu de la Fuente,
leading a team of consultants, junior doctors, senior clinical nurse specialists, psychologists, pharmacists, a dietician and Arabic liaison staff. The team has achieved some of the world’s best outcomes in managing blood disorders, pioneering new bone marrow transplant techniques that are less toxic. “We undertake transplants for children who
either cannot make blood or cannot make the right type of blood,” says Dr de la Fuente. “This is either because they have thalassemia where they cannot make haemoglobin or because they have sickle cell disease, the wrong type of haemoglobin. Some patients have been born with a condition that prevents them from making blood.”
Specialised treatment There is a high incidence of thalassemia and sickle cell disease in people from African, Mediterranean and South Asian backgrounds. Two thirds of the transplants Dr de la Fuente performs are for these conditions, and while they are very common in the UK his patients come from the Middle East and Asia as well, where specialised treatment is not always available. In particular there is a specific form of bone
marrow failure called Diamond Blackfan anaemia, a rare condition where the bone marrow fails to produce red blood cells. These cells are vital because they carry oxygen around the body. Patients are usually diagnosed before the age of one and require ongoing treatment even if transplants are carried out. Dr de la Fuente’s clinic has the largest cohort
of patients in the world for this condition. “We often have to wait until the child is two years old to understand the severity of the disease and to judge whether the transplant is warranted,” he says.
Worldwide donors When bone marrow transplantation was in its infancy the bone marrow was taken from a relative, but one of his innovations was the introduction of bone marrow from volunteer worldwide donors, leading to a greater number of children who could be cured. However the majority of donors tend to be from Western countries and North America, while a number of patients are from ethnic minorities who do not traditionally act as donors. “To get over the fact that most people don’t have a donor we started to use transplants from only one of the parents,” he says. “We now have the technology to do a successful transplant with only a 50 percent match.” With thalassemia Dr de la Fuente can predict early in the condition how severe it is going to be, and
global-opportunity.co.uk
|
Imperial College Healthcare NHS Trust DR JOSU DE LA FUENTE
Dr Josu de la Fuente trained in haematology at Imperial College and Great Ormond Street Hospital achieving membership of the Royal College of Pathologists. He was appointed in 2006 to
lead the paediatric Blood and Marrow Transplant Programme at St. Mary’s Hospital, which specialises in transplantation for haemoglobinopathies and bone marrow failure, and has been innovative in the use of alternative donors. He has also developed a unique clinical and research programme for Diamond Blackfan anaemia patients. He is member of the Scientific Committee UK Thalassaemia Society and is currently the clinical lead for paediatric specialities at Imperial College Healthcare NHS Trust. He is a member and haematology lead in the Paediatric Medicine Clinical Reference Group of NHS England developing national standards of care.
children who require treatment will often come to his Day Unit, though many will also be treated in their own local hospitals. “For sickle cell lysis (the breaking down of cell
membranes) there is a surveillance programme to protect those who are going to have the most severe consequences, and there is a series of criteria that determine how severe the symptoms are. That consultation is for the patients who have bone marrow failure syndrome and cannot make blood. Then we have to wait till the point when the bone marrow cannot sustain the blood production. On average that is around seven years of age but it could be early or later.”
Transplant risk Transplantation has its own risks, so each patient is carefully evaluated to make sure it is the right course of action. Once the decision to transplant is taken, an evaluation period of three months takes place in the Day Unit to check whether the child can cope with the transplant. “We are particularly interested in the collection of ions by transfusions and the harm that that may have caused to the liver or the heart. We also have
Issue 01 | Global Opportunity Healthcare 2015 55
Page 1 |
Page 2 |
Page 3 |
Page 4 |
Page 5 |
Page 6 |
Page 7 |
Page 8 |
Page 9 |
Page 10 |
Page 11 |
Page 12 |
Page 13 |
Page 14 |
Page 15 |
Page 16 |
Page 17 |
Page 18 |
Page 19 |
Page 20 |
Page 21 |
Page 22 |
Page 23 |
Page 24 |
Page 25 |
Page 26 |
Page 27 |
Page 28 |
Page 29 |
Page 30 |
Page 31 |
Page 32 |
Page 33 |
Page 34 |
Page 35 |
Page 36 |
Page 37 |
Page 38 |
Page 39 |
Page 40 |
Page 41 |
Page 42 |
Page 43 |
Page 44 |
Page 45 |
Page 46 |
Page 47 |
Page 48 |
Page 49 |
Page 50 |
Page 51 |
Page 52 |
Page 53 |
Page 54 |
Page 55 |
Page 56 |
Page 57 |
Page 58 |
Page 59 |
Page 60 |
Page 61 |
Page 62 |
Page 63 |
Page 64 |
Page 65 |
Page 66 |
Page 67 |
Page 68 |
Page 69 |
Page 70 |
Page 71 |
Page 72 |
Page 73 |
Page 74 |
Page 75 |
Page 76 |
Page 77 |
Page 78 |
Page 79 |
Page 80 |
Page 81 |
Page 82 |
Page 83 |
Page 84 |
Page 85 |
Page 86 |
Page 87 |
Page 88 |
Page 89 |
Page 90 |
Page 91 |
Page 92 |
Page 93 |
Page 94 |
Page 95 |
Page 96 |
Page 97 |
Page 98 |
Page 99 |
Page 100 |
Page 101 |
Page 102 |
Page 103 |
Page 104 |
Page 105 |
Page 106 |
Page 107 |
Page 108 |
Page 109 |
Page 110 |
Page 111 |
Page 112 |
Page 113 |
Page 114 |
Page 115 |
Page 116 |
Page 117 |
Page 118 |
Page 119 |
Page 120 |
Page 121 |
Page 122 |
Page 123 |
Page 124 |
Page 125 |
Page 126 |
Page 127 |
Page 128 |
Page 129 |
Page 130 |
Page 131 |
Page 132 |
Page 133 |
Page 134 |
Page 135 |
Page 136 |
Page 137 |
Page 138 |
Page 139 |
Page 140 |
Page 141 |
Page 142 |
Page 143 |
Page 144 |
Page 145 |
Page 146 |
Page 147 |
Page 148 |
Page 149 |
Page 150 |
Page 151 |
Page 152 |
Page 153 |
Page 154 |
Page 155 |
Page 156 |
Page 157 |
Page 158 |
Page 159 |
Page 160 |
Page 161 |
Page 162 |
Page 163 |
Page 164 |
Page 165 |
Page 166 |
Page 167 |
Page 168 |
Page 169 |
Page 170 |
Page 171 |
Page 172 |
Page 173 |
Page 174 |
Page 175 |
Page 176 |
Page 177 |
Page 178 |
Page 179 |
Page 180 |
Page 181 |
Page 182 |
Page 183 |
Page 184 |
Page 185 |
Page 186 |
Page 187 |
Page 188 |
Page 189 |
Page 190 |
Page 191 |
Page 192 |
Page 193 |
Page 194 |
Page 195 |
Page 196 |
Page 197 |
Page 198 |
Page 199 |
Page 200 |
Page 201 |
Page 202 |
Page 203 |
Page 204