The report is available in English with a French summary - KCE

KCE Reports 120 Plasma 83
• For bone marrow or stem cell transplant, the frequency of administration
averaged 10.8 per year, ranging 2.6-12.9. In general in children, treatment
is administered before transplant and during at least 3 months, mostly 6
months and up to 1-2 years in cases of persistent B-cell abnormalities.
However, the recent decrease in myeloablative transplants, due to an
increase in non-myeloablative techniques, has decreased the level and
duration of secondary immune deficiency - and the consequent IG needs.
This is mostly described in adult patients.
• For ITP, the frequency of administration was not available in most
hospitals but was 1.4 per case in average in 3 hospitals.
• In Kawasaki disease, the frequency of administration could not be
established based on available data, but experts advised the administration
of a single dose, followed by a 2nd dose in a few patients in which
inflammatory signs and symptoms are still present.
WHICH CASES SHOULD BE TREATED WITH IG
The proportion of patients receiving IG for each of these indications in Belgium was
difficult to assess in this survey, as clinicians could not always collect data on the total
number of cases that visited their centre.
For bone marrow or stem cell transplant, paediatricians considered that nearly all
paediatric cases require IG supplementation; in adults, only a selected subgroup of
patients having a symptomatic secondary immunodeficiency following the transplant can
benefit from IG treatment, and this should be decided on an individual basis.
Secondary immune deficiencies due to the use of therapies depleting B-cells or impairing
B-cell response (eg. some anticancer drugs) and inducing symptomatic
hypogammaglobulinemia are also medical conditions for which IG may be necessary.
Experts proposed that cases with secondary hypogammaglobulinemia be treated with IG
during for the period of the hypogammaglobulinemia, irrespective of its cause. This is
line with the approach of PID cases, though the treatment would be mostly limited to a
period of 6-12 months.
ESTIMATION OF AMOUNTS
The amount of IG used for each immuno-haematological indication in these centres has
been estimated, based on the assumption of an average 75kg body weight per adult, 25
kg per child and 4 kg per neonate. Under these assumptions, treatment of PID patients
would represent 61% of the total IG use for immuno-haematological indications, CLL
and MM 14% and transplants 10%. However, these estimates are biased by a better
response rate among paediatricians as compared to haematologists treating adult cases.
USE OF SUB-CUTANEOUS IG
Subcutaneous administration of IG was used for PID in 5 out of the 6 hospitals and used
to treat 44% of the PID paediatric cases in average. This practice was predominant in
two hospitals where 48-71% of PID children received SC administration, and is a
growing practice in the other paediatric wards of these centres. Expert paediatricians
consider that SC administration is a first choice for long-term treatment as it improves
quality of life of the patient and is more effective to control immunodeficiencies, as IG
levels are better maintained with weekly infusions. In adult cases however, only two out
of four hospitals were using SC administration, and this concerned 14 and 44% of the
PID adult cases.
In neurological diseases
In order to know the current use of IG in Belgium for the neurological and neuromuscular
indications consuming large amounts of IG, an expert meeting was organized.
The criteria for IG treatment, proportion of cases treated, dosage and frequency of IG
treatment were discussed, based on information found in the scientific literature and
the Belgian situation. The survey also collected data on 284 IG administrations for
neurological cases (Guillain Barré disease, CIDP and MMN).