New England Journal of Medicine JUNE 12, 2003

Prevention of Recurrent Preterm Delivery by 17 Alpha-Hydroxyprogesterone Caproate

Paul J. Meis, Mark Klebanoff, Elizabeth Thom, et al. for the National Institute of Child Health and Human Development Maternal-Fetal Medicine Units Network

Bottom Line

In women with a prior spontaneous preterm birth, weekly administration of 17 alpha-hydroxyprogesterone caproate (17P) significantly reduces the risk of recurrent preterm delivery compared to placebo.

Key Findings

1. 17P treatment significantly reduced the primary outcome of delivery at less than 37 weeks gestation (36.3% in the progesterone group vs. 54.9% in the placebo group; relative risk [RR] 0.66; 95% CI, 0.54-0.81).
2. The risk of delivery at less than 35 weeks was significantly reduced with 17P (20.6% vs. 30.7%; RR 0.67; 95% CI, 0.48-0.93).
3. The risk of delivery at less than 32 weeks was also significantly lower (11.4% vs. 19.6%; RR 0.58; 95% CI, 0.37-0.91).
4. Infants born to mothers in the 17P group experienced significantly lower rates of necrotizing enterocolitis, intraventricular hemorrhage, and the need for supplemental oxygen.

Study Design

Design
RCT
Double-Blind
Sample
463
Patients
Duration
up to 36 wk
Median
Setting
Multicenter, US
Population Pregnant women with a documented history of a prior spontaneous preterm delivery.
Intervention Weekly intramuscular injections of 250 mg of 17 alpha-hydroxyprogesterone caproate (17P) starting at 16-20 weeks gestation until delivery or 36 weeks.
Comparator Weekly intramuscular injections of an inert oil placebo.
Outcome Preterm delivery before 37 weeks of gestation.

Study Limitations

The placebo group's high recurrence rate (54.9%) led to persistent scientific debate regarding whether the observed risk reduction was inflated, potentially due to the specific characteristics of the enrolled population or the vehicle (castor oil) used in the injections.
The study did not explore dose-response relationships, and subsequent research has suggested that variability in drug concentrations may influence treatment efficacy.
The study was limited to a specific high-risk population (prior spontaneous preterm birth), which may not generalize to other etiologies of preterm birth.

Clinical Significance

This trial established 17-hydroxyprogesterone caproate as the standard of care for the prevention of recurrent spontaneous preterm birth in women with a history of prior preterm delivery, significantly impacting obstetric practice and neonatal morbidity reduction for two decades.

Historical Context

Prior to this landmark trial, evidence supporting progesterone therapy for preterm birth prevention was inconsistent and derived from small, disparate studies. The Meis trial provided the first robust, multicenter randomized controlled evidence, which led to widespread clinical adoption and established 17-OHPC as a primary preventive strategy in high-risk pregnancies.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the biological role of progesterone in maintaining pregnancy, and why was 17-alpha-hydroxyprogesterone caproate (17P) hypothesized to prevent recurrent preterm birth?

Key Response

Progesterone is essential for maintaining uterine quiescence. It inhibits the production of pro-inflammatory cytokines and prostaglandins, decreases the expression of contraction-associated proteins, and modulates the immune response at the maternal-fetal interface. 17P is a synthetic progestogen that mimics these effects to delay the onset of labor in women with a history of spontaneous preterm delivery.

Resident
Resident

According to the Meis trial, what are the specific criteria for patient selection and the recommended timing for initiating 17P therapy to reduce the risk of recurrent preterm birth?

Key Response

The study included women with a singleton pregnancy and a documented history of a previous spontaneous preterm delivery (defined as delivery between 20 and 36 weeks 6 days). Treatment should be initiated between 16 weeks 0 days and 20 weeks 6 days of gestation and continued weekly until 36 weeks 6 days or delivery.

Fellow
Fellow

The Meis trial demonstrated a reduction in delivery at <37, <35, and <32 weeks. How does the magnitude of the effect on neonatal morbidity, specifically rates of intraventricular hemorrhage and necrotizing enterocolitis, inform the clinical value of 17P beyond just prolonging gestation?

Key Response

The trial found significant reductions in several neonatal morbidities: necrotizing enterocolitis was reduced from 2.7% to 0%, and intraventricular hemorrhage from 5.2% to 1.3%. This suggests that 17P does not merely delay birth slightly but significantly improves neonatal survival and quality of life by preventing early-gestation deliveries where these complications are most prevalent.

Attending
Attending

How do you reconcile the robust positive results of the 2003 Meis trial with the subsequent negative results of the 2019 PROLONG trial when counseling a high-risk patient today?

Key Response

The Meis trial was conducted in a higher-risk population with a 54.9% preterm birth rate in the placebo group, whereas the PROLONG trial (Study 003) had a much lower placebo rate (approx. 11%). This suggests that 17P's efficacy may be most pronounced in very high-risk populations, though the FDA ultimately withdrew approval because the confirmatory trial failed to replicate the clinical benefit in a modern, lower-risk cohort.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The Meis trial utilized a 2:1 randomization scheme (treatment to placebo). What are the specific implications of this allocation ratio regarding the statistical power for secondary outcomes and the potential for detecting rare adverse events?

Key Response

A 2:1 ratio increases the number of participants exposed to the active drug, enhancing the safety profile database and potentially improving recruitment. However, for a fixed total sample size, it reduces the statistical power to detect differences in the primary outcome compared to 1:1 randomization. It also complicates the detection of rare side effects if those effects are similar to baseline pregnancy complications.

Journal Editor
Journal Editor

As a reviewer, the 54.9% preterm birth rate in the placebo arm of the Meis trial stands out as exceptionally high. To what extent does this 'high-risk' baseline limit the external validity of the study for the general obstetric population in the United States?

Key Response

An unusually high event rate in the control group can lead to an overestimation of treatment effect (Absolute Risk Reduction) when applied to the general population. Editors would flag this as a threat to generalizability, as the study population—largely recruited from academic centers with high rates of poverty and prior PTB—might not represent the standard risk profile seen in community practice.

Guideline Committee
Guideline Committee

Given that the Meis trial was the primary evidence for ACOG and SMFM recommendations for nearly two decades, how should committees pivot following the 2023 FDA withdrawal of Makena (17P)?

Key Response

Committees must now emphasize that while the Meis trial was methodologically sound for its time, the failure of the confirmatory PROLONG trial indicates that 17P lacks a consistent clinical benefit across modern populations. Current guidelines (ACOG Practice Advisory) have shifted toward recommending vaginal progesterone for those with a short cervix and de-emphasizing 17P for history-indicated prophylaxis.

Clinical Landscape

Noteworthy Related Trials

2011

PREGVID Trial

n = 657 · Ultrasound Obstet Gynecol

Tested

Vaginal progesterone 200mg daily

Population

Asymptomatic women with a short cervix

Comparator

Placebo

Endpoint

Preterm birth before 34 weeks of gestation

Key result: Vaginal progesterone significantly reduced the rate of preterm birth in women identified with a short cervix on ultrasound.
2016

OPPTIMUM Study

n = 1,228 · Lancet

Tested

Vaginal progesterone 200mg daily

Population

Women at risk of preterm birth

Comparator

Placebo

Endpoint

Composite of perinatal mortality, morbidity, and neurodevelopmental impairment

Key result: Vaginal progesterone did not significantly improve perinatal outcomes compared to placebo in the studied population.
2018

IMPROVE Study

n = 900 · AJOG

Tested

Vaginal progesterone 200mg daily

Population

Women with prior preterm birth and singleton gestation

Comparator

Placebo

Endpoint

Preterm birth before 37 weeks

Key result: Progesterone demonstrated a reduction in recurrent preterm birth among high-risk patients, supporting the benefit seen in earlier landmark studies.

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