Long-term outcomes in five patients with Autoimmune pulmonary alveolar proteinosis treated with Molgramostim Inhalation Solution

14 evidence topics · 1 source

Objective

Long-term treatment outcomes

“long-term effects of molgramostim inhalation solution” (opens the source at this quote in a new tab)

Location and study date

Case-specific treatment initiation dates

Case	Reported initiation of molgramostim
Case 1	“June 2019”
Case 2	“March 2020”

Study design

Observational case series

“real-world case series” (opens the source at this quote in a new tab)

Eligibility criteria

Diagnostic findings in the reported cases

Finding	Case 1	Case 2	Case 3	Case 4	Case 5
Anti-GM-CSF antibody	“Positive”	“Positive”	“Positive”	“Positive”	“Positive”

Prospectively defined eligibility criteria

No evidence found.

Treatment

Compassionate-use inhalation treatment

“vibrating mesh nebuliser” (opens the source at this quote in a new tab)
“compassionate-use programme” (opens the source at this quote in a new tab)

Reported duration of molgramostim exposure

Case	Time on molgramostim, years
Case 1	“4.5”
Case 2	“3.5”
Case 3	“5.5”
Case 4	“4 and previous 1 year in IMPALA trial”
Case 5	“1.5 and previous 1 year in IMPALA trial”

Study outcomes

Rationale for using DLCO percent predicted as an observational outcome

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

Reported case count

Population	Reported number
Participants in the case series	“five”

Description of analysis sets

Retrospective data collection

“retrospective nature” (opens the source at this quote in a new tab)

Formal comparative analysis sets

Not applicable.

Results

Participant disposition

Ages at last reported follow-up

Case	Age at last follow-up, years
Case 1	“31”
Case 2	“33”
Case 3	“40”
Case 4	“32”
Case 5	“52”

Baseline characteristics

Ages at presentation as reported in the case-series table

Case	Age at presentation, years
Case 1	“27”
Case 2	“29”
Case 3	“31”
Case 4	“21”
Case 5	“46”

Efficacy results

Pulmonary function before treatment and at last assessment

Measure, initial/last	Case 1	Case 2	Case 3	Case 4	Case 5
DLCO percent predicted	“37/91”	“36/60”	“39/87”	“62/90”	“12/44”
Forced vital capacity percent predicted	“64/74”	“83/122”	“52/71”	“73/88”	“54/75”

Health-related quality of life and patient-reported outcomes

Case 5: return to employment

“he has returned to work.” (opens the source at this quote in a new tab)

Standardized patient-reported outcome scores

No evidence found.

Safety results

Reported serious adverse events

“There were no reported serious adverse events in these five patients while receiving molgramostim and the treatment has been well tolerated.” (opens the source at this quote in a new tab)

Study limitations

Summary

This retrospective series describes five participants without a concurrent comparator. Treatment exposure and follow-up duration varied, and two cases included preceding IMPALA exposure. The reported pulmonary-function improvements cannot establish a comparative treatment effect. Absence of reported serious adverse events in five cases does not estimate the frequency of uncommon harms, and the qualitative return-to-work report is not a standardized quality-of-life assessment.