Clinical trial NCT05077150: A Case-control Study on Risk Factors, Timing, and PCR Use, for Pneumocystis Pneumonia (PcP) After Allogeneic HSCT
Official title: A Case-control Study on Risk Factors, Timing, and PCR Use, for Pneumocystis Pneumonia (PcP) After Allogeneic HSCT
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pneumocystis Pneumonia
Keywords: jirovecii
pneumocystis
PCP
Lead sponsor: European Society for Blood and Marrow Transplantation
Sponsor class: NETWORK
First posted: 2021-10-14
Enrollment: 168
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients who have received an allogeneic HSCT during the last 24 months and who have a BAL fluid positive for Pneumocystis jirovecii (qPCR or IF or cytology) during the study period will be included, irrespectively of age, transplant characteristics and irrespectively to the fact that the patient has been treated for PcP or not.

Assuming an incidence of 3% after allogeneic HSCT, a total number of 3 300 allogeneic transplant (roughly 100 centers) would allow to expect 100 cases of PcP.

Brief summary: The fungus Pneumocystis jirovecii is responsible for pneumocystosis (PcP), a life threatening pneumonia in patients undergoing HSCT. The spontaneous attack rate of 16% within the first 6 months following allogeneic HSCT reported in the 1980's has considerably decreased with prophylaxis. However, PcP still remains a concern in the transplant ward with an incidence rate up to 2.5% in allo- and 1.4% in autologous HSCT but up to 7.2% on low dose of Dapsone. The mortality of PcP is especially high in HSCT recipients. One of the main factors of PcP after HSCT seems to be either the lack of TMP-SMX prophylaxis (all the other prophylactic drugs being inferior to TMP-SMX), or poor compliance to prophylaxis. Due to the rarity of the disease after HSCT, it is impossible to study it in monocenter studies, except on very long periods of time which may not reflect current practice. Several questions deserve investigations in a multicenter study, about timing, risk factors, and outcome.

Moreover, some European laboratories involved in the diagnosis of PcP have already given up to classical diagnostic methods and switched to qPCR. This implies that lower fungal burden can be detected and the clinical pertinence of such a diagnostic strategy deserves to be assessed.

Detailed description: Due to the lack of standardization, qPCR on sputum only will not be taken in account for the diagnosis of PcP. Knowing this is a non-interventional study, no additional visits or laboratory tests will be performed for the study. Only the available data will be collected.

Eligibility criteria: Inclusion Criteria PCP cases:

* Allogeneic HSCT within the previous 24 months
* New case (first onset) of PcP documented in a BAL fluid, whatever the positive diagnostic test (cytology or IF or PCR) and whatever the presentation and treatment
* Any age
* Pre or post-transplant signed informed consent to enter the data in the EBMT registry

Exclusion Criteria PCP cases:

* Autologous HSCT
* Allogeneic HSCT recipient transplanted more than 24 months at time of the onset of PcP
* Second episode of PcP since allogeneic HSCT (patients who had experienced PcP before the allogeneic HSCT are not excluded).

Primary outcomes:
measure: Post-transplant risk factors for PCP infection
timeFrame: 90 days
Secondary outcomes:
Other outcomes:
Locations:
city: Leuven
country: Belgium
facility: University Hospital Gasthuisberg
city: Amiens
country: France
facility: University of Amiens: CHU Amiens
city: Creteil
country: France
facility: Hôpital Henri Mondor
city: Lille
country: France
facility: Hôpital Huriez
city: Paris
country: France
facility: Hopital St. Louis
city: Hamburg
country: Germany
facility: University Hospital Eppendorf
city: Budapest
country: Hungary
facility: United St. Istvan and St. Laszlo Hospital
city: Haifa
country: Israel
facility: Rambam Medical Center
city: Oslo
country: Norway
facility: Oslo University Hospital, Rikshospitalet
Study officials:
affiliation: Hematology Department, Pr Cordonnier. henri Mondor University Hospital
name: Christine Robin, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Paediatric Haematology Oncology. Policlinico G.B. Rossi
name: Simone Cesaro, MD
role: STUDY_CHAIR
References: